Update National Liver Review Board Guidance Documents and Policy Clarification At a glance Current policy The National Liver Review Board (NLRB) reviews requests from transplant programs for candidates whose model for end-stage liver disease (MELD) score or pediatric end-stage liver disease (PELD) score does not reflect their medical urgency for transplant. The NLRB uses policy and guidance documents to inform their decision to approve or deny a request for an exception score. Supporting media Presentation View presentation slides Proposed changes Include one additional diagnostic criteria for a candidate to receive an automatic exception for cholangiocarcinoma (CCA) in OPTN policy. Update NLRB guidance documents for pediatric exceptions, candidates with a neuroendocrine tumor (NET), and candidates with primary (PSC) or secondary sclerosing cholangitis (SSC). Anticipated impact What it's expected to do Provide NLRB members with updated guidance to use when reviewing exception score requests for specific candidates Provide transplant programs with an updated list of diagnostic criteria when submitting a standardized exception request for a candidate with CCA What it won't do Will not impact how liver candidates are prioritized on a match run Themes National Liver Review Board (NLRB) improvements Terms to know Candidate: An individual on the organ transplant waiting list Transplant program: An organ specific facility within a transplant hospital Guidance Documents: Documents that provide more information to transplant programs and NLRB members to use when making decisions on exception requests Model for End-Stage Liver Disease (MELD): The scoring system used in allocation of livers to candidates who are at least 12 years old Pediatric End-Stage Liver Disease (PELD): The scoring system used in allocation of livers to candidates who are under 12 years old National Liver Review Board (NLRB): A review board of members drawn from a nationwide pool of liver transplant physicians and surgeons, who review exception requests from transplant programs for candidates whose automatically calculated model for end-stage liver disease (MELD) score or pediatric end-stage liver disease (PELD) score does not accurately reflect the candidate’s medical urgency for transplant. Standardized exception: A exception with criteria outlined in policy that is automatically approved when submitted and is not reviewed by the NLRB Cholangiocarcinoma (CCA): Bile duct cancer Neuroendocrine Tumor (NET): A tumor that forms in the liver Primary Sclerosing Cholangitis (PSC): A rare disease that causes scarring in the bile ducts Secondary Sclerosing Cholangitis (SSC): A chronic disease similar to PSC caused by a known condition Click here to search the OPTN glossary Comments Anonymous | 03/24/2021 I would like to express my strong support for the proposed modifications to the NLRB policy as they relate to PSC. For too long victims of this rare disease (a disease of unknown origins that is not caused by anything they did or lifestyle choices they made) have had to stand aside, getting sicker and sicker, their liver not functioning, because this disease does not produce the numbers needed to be placed appropriately on the transplant list. This proposal is a step in the right direction to lower the bar for PSC patients to be granted exceptions and resulting in more equitable organ transplant. My daughter was diagnosed with this disease when she was 12. She managed the disease well for over 20 years but now, in a 6-month period, she has gone from leading a relatively normal life to being almost completely bedridden with multiple hospitalizations. We watch her getting sicker and weaker, in pain with an expanding list of symptoms, while her MELD scores don't hit the magic numbers. This young woman and her 2 YO daughter deserve to not be held hostage by an antiquated "scoring system" that favors a certain type of liver disease over others. Joseph Panessiti | 03/23/2021 Please allow exception points for PSC/PBC patients. I waited for 18 months on the list while living donors were evaluated. Shortly after one was found, my MELD increased from 23 to 30. Two weeks later it was 36, and a week after that I received a new liver from a deceased donor. While I was in recovery, the surgeon told my wife that my liver was so bad, he didn't think I would have survived more than a few days without the transplant. This is a common story among PSC & PBC patients. I wasn't surpised when I found out (a week or so after surgery) because I'd already heard others share similar experiences. Please help save more lives by allowing exception points to better reflect the candidate's status! Anonymous | 03/23/2021 When my Husband was diagnosed with PSC at the age of 29, after managing Crohn's since he was 13, we had no idea what we were up against. A constant temporary fix, or band-aid, in the form of procedures and surgeries, just so he could get by. With every fix, his MELD would decrease a bit, until the next bile duct blockage and then it would sky rocket. Is this how his life was going to have to be? Waiting for his PSC to cause liver cancer or getting so weak until his MELD score was high enough to be considered for a transplant? So weak, he possibly could not even endured a transplant and a challenging recovery? By the grace of God, an altruistic donor came forward on July 14th, 2020 and offered a portion of their liver for my husband. So many others, do not have that opportunity. They wait and wait and wait. Getting weaker and weaker. We strongly support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. People with PSC deserve this chance. They didn't drink their way to liver disease. They have absolutely no say in it. They live with it every single day, aware of there not being a cure. They want to live the best life possible for themselves and for their families. Please help so many PSC warriors with MELD exception points. They deserve a fighting chance at a quality life. Thank you! Don Safer | 03/23/2021 I would like to add my strong support for the changes proposed by the NLRB for PSC patients. Thank you for giving some hope to the PSC community. Maritza R Pratt | 03/23/2021 I am in support of the policy change for exception points for PSC patients. This change could help the sickest and desperate patients. Two of my dear friends are transplant recipient and because of recurrent PSC one of then is a probable future candidate for a second transplant, the other one just passed away. Shortly after my first friend was diagnosed with recurrent PSC, she was hospitalized with an infection. In that hospitalization and the follow ups with her doctor , they recognized that she will eventually need a transplant and probably wouldn’t qualify for another deceased donor via MELD. I am very concern that she may not make it to transplant, she is very sad, depress and worry about her future. She is an asset to the society because of all the excellent volunteer work she does in support of PSC moms and her many creative talents in the Hispanic community. She is a mother, wife, sister, daughter and aunt to a big family of friends, too. We will love to have it among us for many years to come. Please give her the gift of life, in the name of Jesus Christ, that gave His life and blood for all of us. This is not a political issue, this are real peoples. Thank you very much. Julianne Vasichek | 03/23/2021 I strongly support any policy change that would make more exception points for the MELD score available to patients with PSC. Unfortunately, I have watched many of my friends and PSC community members pass while waiting for transplant or be removed from the list due to decompensation or other conflicting factors. My comment today is regarding a friend of mine who has successfully made it through two liver transplants, both due to PSC, and will likely need a third hopefully much further down the road. Prior to my friend's second transplant I was able to spend more time with her, because I was recovering from my own transplant and subsequent intestinal surgeries. This put us in Rochester together for weeks or months at the same time. I spent more time than I would like to admit, because you try not to be scared for your friends, wondering if she would make it to her second transplant. Just about 3-4 weeks before her transplant happened, I spent time with her before flying home for Christmas. I honestly was not sure if I would see her after Christmas. One of the most stark memories I have from that time was from the table in the dining room. Her hepatic encephalopathy was so severe that it would often take her 1-2 hours to eat because she kept falling asleep. She was already sleeping for the majority of the day. I would sit across from her and jokingly say her name or ask her a question as she started to drift off, spoon or cup in hand. One day I sat next to her and tapped her on the shoulder or nudged her every time she would start to fall over with either a spoon of food or with food in her mouth. She would fall asleep within a minute of being "awake." It was utterly frightening and almost incomprehensible because she needed the nutrition, but could not stay conscious to eat without help. Her MELD score was in the mid-20's at this time. I remember when she would experience a complication that positively, meaning added points, to her MELD score she would cringe and deal with the effects, but be slightly excited because it would increase her chance for transplant for that week. She teetered on the same line I had seen other friends walk, some making it through to transplant, and some succumbing to either PSC or a complication which took them off the list or ultimately took their life. I cannot recount how many times I have heard a fellow PSC'er say after transplant, that walked the thin MELD line, "the surgeon was amazed at how my bad my liver was when they finally saw it." Months and years of decompensation a lot of times leave some of the strongest people I have seen with little physical strength to fight complications. Please support the policy changes which would count more infections during hospitalizations, a common occurrence in end stage liver disease, into the MELD Score. Right now, our community members are hanging on in hopes that somehow they will "get sick enough in the right way for their score to increase." It just doesn't make sense. Julianne James Leach | 03/23/2021 I strongly support the proposed changes regarding the exception points for PSC patients. Anonymous | 03/23/2021 I am the father of a young adult who was diagnosed with PSC 15 years ago. I support the proposed changes to the NLRB policy. Currently, transplant is the only treatment available for PSC. We have known too many fellow patients and dear friends die waiting for a transplant. Thank you, NLRB, for proposing data-based changes that recognize how PSC and other liver diseases differ in their progression and severity, and how that can be used to improve the current MELD-based allocation system. Improving the allocation process will save lives! Thank you. Anonymous | 03/23/2021 Our son received a life saving liver transplant in 2015. He had been ill for 17 years. PSC, which caused: Portal hypertension, varicees, infarcted spleen, stomach perforation, appendectomy and more. In 2015 he was in and out of the hospital with cholangitis attacks. His MELD was not high enough for a liver transplant despite how sick he was. PSC is a terrible disease. Please support exception points for this disease. Anonymous | 03/23/2021 I am expressing my strong support for the proposed modifications to the NLRB policy, specifically as it relates to PSC. I specifically was diagnosed with PSC 12 years ago, but in the last year have seen my meld fluctuate to as high as 24 and dip down to 15, I have been hospitalized on numerous occasions and was only put on the transplant list recently partially due to the high meld needed for transplantation without any exception points. I have now been listed on the transplant list, but do am uncertain if/when I will get a call for a transplant before it is too late. Anonymous | 03/23/2021 I strongly support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My son has received 4 liver transplants in the past 17 years. I have watched him suffer waiting for his MELD to get high enough to receive a transplant. His 4 th transplant he withered away for 2 years on the transplant list with multiple tubes draining from his abdomen. The MELD score is unfair to those that suffer from PSC. Lindsey Nanz | 03/23/2021 I very strongly support the proposed update to the NLRB guidance documents and the policy clarification. I am 25 years old, and by age 21 I had already received two liver transplants due to PSC and recurrent PSC. After five months of being on the waiting list for my first liver transplant (which I received at age 15), I had a life-threatening variceal hemorrhage that led to vomiting blood before I fell into a coma for three days. My transplant doctor told my parents that I wouldn't live to see the top of the transplant list and that we had to line up a living donor as soon as possible. Under the regional review board exception system, my doctors appealed at least five times for exception points but were never granted any. My mom, two of my brothers, and my cousin went through the exhaustive living donor evaluation process. It was sheer luck that I received a living donor (domino) transplant after ten months on the waiting list. Unfortunately, I never felt better, my quality of life decreased, and my PSC returned two years later. As my PSC progressed, I experience the "suicidal itch." For four years, my itching was uncontrollable and I tried many medications and treatments with very little success. Between my first and second transplants, I was hospitalized about 25 times with serious liver infections - but my capable hospitals did not need to put me in the ICU to treat them. Because of the infections, I had PICC lines placed 10 times, until my veins were no longer any good, and then I had a port placed, which got me through the next five years of hospitalizations (second transplant included). These hospitalizations prove that one can be in serious end-stage liver failure without ever being admitted to the ICU! When I needed to be listed the second time, at age 21, I was listed in two regions (as a pediatric in one and as an adult in the other). Both regions were under the regional review board system. This time, BOTH regional boards recognized that I was extremely ill, despite a MELD score of just 12, and both awarded me a MELD score of 30. Once at MELD 30, I still waited five months before receiving a deceased donor transplant. I was quickly deteriorating, dropping weight rapidly, losing strength, etc. I knew I was dying. I received my second transplant in 2017, but under the 2019 NLRB policies, I would not have been awarded exception points and might never have received a transplant. The new proposal is an important step that would help many PSCers, but we urge you to continue to examine the data carefully. I am very involved in the PSC community and have had too many friends die because of this awful disease. I know that PSCers can get very, very sick while holding a MELD in the teens or low 20s. If they catch an acute infection, they don't always survive it because they are too weak. These patients are on the waiting list due to the bad luck that is autoimmune disorders and not because of poor life choices, and at this time a transplant is the only real treatment for PSC. I am so grateful that you are listening to the PSC community and proposing these changes. Please continue to keep us in mind when reviewing guidance documents. Anonymous | 03/23/2021 Our daughter was diagnosed at age 16 with PSC. We are deeply concerned for her health/future and for the health of the other members of the PSC community. We are writing to strongly support the proposed policy changes regarding exception points be added to the MELD scores for PSC patients. From what we have learned/observed, it is clear that there is a serious discrepancy between MELD scores of PSC patients and how seriously ill they may be. It is disheartening to learn of PSC patients who suffer on wait lists with moderate MELD scores, often becoming too sick to receive a transplant. We are hopeful for policy change that will recognize the science/research and ultimately help save and improve lives for these patients with this orphan disease. Many thanks for considering this change. It will provide greater hope/reassurance for the future of our daughter and the rest of the grateful PSC community. Anonymous | 03/23/2021 I am writing on behalf of my nephew who is a PSC sufferer; and for his family who loves, supports, and helps him manage his disease daily. It is essential that reassessment of the criteria for treatment occurs as new data is recognized. Removing barriers from potential life-saving treatment is necessary to increase the health and well-being of the patients and for hope for the entire family. I support the proposed modifications to the NLRB policy in relation to PSC sufferers who are waiting for liver transplants. The changes could help PSC patients and their families to maintain hope during the severe health crisis that a PSC patient on the waiting list represents. Ricky Safer | 03/23/2021 I am extremely grateful to the NLRB for suggesting this policy change for PSC patients awaiting transplant. It is an important first step that will take away a layer of hurdles and that will bring some hope to PSC patients struggling to survive while on the transplant list. I am the founder and CEO of PSC Partners Seeking a Cure, the patient organization whose mission is to provide education and support to PSC patients, families, and caregivers, as well as to raise funds to research causes, treatments, and a cure for primary sclerosing cholangitis. Transplant eligibility is a major stress factor that every PSC patient lives with, starting at diagnosis. In the seventeen years since my diagnosis, I have witnessed the journeys of hundreds of PSC patients, and as a PSC patient myself, I have experienced the full weight of living with this disease. And like many others, during the years, I have added a host of other comorbidities to PSC. I watch others and experience myself the unpredictable progression of PSC and intimately know what it means to be living with the threat of looming cholangiocarcinoma, colon cancer and liver transplant. These fears are compounded by the knowledge that as PSC patients, the current MELD score is no reflection of disease severity. The fear and anxiety of knowing that we may not meet the MELD score and other criteria while being extremely sick, take a huge toll on all PSC patients and their families. At PSC Partners, we tirelessly seek alternatives to transplant, our only means of survival, and focus on finding new therapies for this disease. We are afflicted by this devastating disease, not due to any unhealthy personal lifestyle choices. We are a tight knit patient community, and it is painful to watch helplessly as we continue to lose community members who pass away because their MELD scores are not high enough to receive a donor liver or because they have had to wait so long that they are no longer healthy enough to survive a transplant. It is difficult to watch patient families struggle with this life or death situation, especially with children and young adults. The psychological impact on entire families of fighting this physical and emotional battle is overwhelming. It is equally shattering to hear many hepatologists tell their PSC patients that they need to start the process of finding a live donor since their MELD score precludes them from receiving a cadaveric liver transplant in time. The wrenching ethical decision of accepting a loved one to risk their life for us is unacceptable to many patients and families. We appreciate that the NLRB acknowledges the urgency of our situation and is offering us this policy change. I hope that the NLRB will continue its surveillance of wait list monitoring and dropout rates after these changes. Anonymous | 03/23/2021 My husband Rick has been diagnosed with PSC for 17 years. We are starting our transplant evaluation because of his recurrent hospitalizations due to cholangitis and sepsis. His MELD score is low and does not reflect the physical, emotional, and mental anguish this disease causes the patient and family. He has to have an ERCP every 2-3 months in an attempt to avoid sepsis and death. He requires hospital stays with almost every ERCP. Through the innumerable hospitalizations in his lifetime, he rarely has a significant enough change of lab values to adjust his MELD and therefore is not offered an opportunity for liver transplant. PSC symptoms can be severe and relentless and cause significant disability and affects many patients during a time of life they are trying to go to college, start careers and raise children. PSC patients are brave, vibrant, hard-working, courageous, resilient and compliant patients who don't have the energy to accomplish all their goals because the disease doesn't have treatment and eligibility for liver transplant currently doesn't reflect how sick the patient is. After 17 years, Rick is very aware of when it is time to be seen in the emergency department for admission. He is proactive and we know enough to try and get started on IV antibiotics prior to severe bacteremia requiring ICU. As a family physician myself, who has been taught and believes strongly in treating the whole person rather than just the labs or imaging results, it is frustrating and unacceptable to see how PSC patients, caregivers, and their medical teams have to watch someone deteriorate waiting to be "sick enough" per a MELD calculation to be considered for the only possible treatment that a PSC patient is offered for survival. Anonymous | 03/23/2021 I strongly support the allowance of exception points to be added to the MELD scores for PSC patients. The MELD system is terribly unfair to PSC patients, who despite having no control of their progressive disease, are penalized as transplant candidates. My nephew was diagnosed as a young child and we hope that if he needs to be considered for transplant at some point in his life, that he will be able to get one without the risk that he will be made to wait until he is too sick to be considered. Anonymous | 03/23/2021 I am a PSC patient and was recently (January 2021) diagnosed with end stage liver disease. This stage of PSC is still new to me, but in reading the support groups and literature, it appears the current methodology for determining MELD score may not properly factor in certain unique characteristics of PSC. This could lead to a reduced chance of me receiving a liver transplant in a timely manner. I am 42 years old with a wife and 5 young children who I adore and want to be with for many years. I hope when making your decision you factor in the uniqueness of PSC as a disease and approve the addition of MELD exception points for patients like myself. Thank you for your consideration! Brian Carrie Leach | 03/23/2021 As a parent of a child with PSC I strongly support the proposed changes and clarifications regarding exception points for PSC patients. The MELD scores of PSC patients waiting for transplant often do not reflect the severity of their disease. As a result many PSC patients wait on the list and become too sick for transplant. These changes will help those who suffer with PSC get the liver transplant they need. Nathan Baggett | 03/23/2021 I strongly support the proposal to revise how exception points are granted to PSC patients. Prior to my living donor liver transplant, I struggled with recurrent episodes of cholangitis that required hospitalization. These infections seemingly become more severe over time. I received a live donor liver transplant in 2017 after being on the transplant waiting list for nearly two years. During that time on the waiting list, despite getting sicker and sicker, my MELD score only slowly increased. I faced mounting complications, but my MELD score was never high enough to garner an organ offer. My doctors made clear to me that my only hope was a living donor. My MELD score never reflected how sick I really was, and my family and I worried that I would eventually become to frail to be transplanted before my MELD score reached a point that would qualify me for an organ offer. The proposed change reflects the true reality of the severity of PSC for many patients and allows PSC patients a better opportunity to access a life saving transplant. This proposed change would give PSC patients an equitable shot at receiving an organ transplant. Sonia Lugo | 03/23/2021 My daughter is a transplant recipient and because of recurrent PSC will probably need another. I am in support of the policy change for exception points for PSC patients. She has been hospitalized with Bacteremia in just the first year since diagnosis of rPSC. She will eventually need a transplant and has already been told probably wouldn’t qualify for another deceased donor via MELD. I support any change that will give PSCers like my daughter a chance at transplantation that the current system does not give. Rob Mottola | 03/23/2021 I am newly diagnosed with PSC CIRRHOSIS PORTAL HYPERTENSION and UC. This has been a crushing blow to me my wife and 4 kids. Please support the exceptions proposal and save more lives. Thank you. Anonymous | 03/23/2021 I strongly support the allowance of exception points to be added to the MELD scores for PSC patients. The MELD system is extremely unfair to PSC patients, and despite having no control of their progressive disease, they are penalized among all transplant candidates. My nephew was diagnosed as a young child and we hope that if he needs to be considered for transplant at some point in his life that he will be able to get one without risk that he will be made to wait until he is too sick to be considered. Region 10 | 03/23/2021 Region 10 sentiment: 2 Strongly Support, 12 Support, 2 Neutral/Abstain, 0 Oppose, 0 Strongly Oppose. Comments: In regards to PELD exceptions, one member expressed concerned at the time frame for 2 admissions per year. Their suggestion would be to shorten the time frame between admissions. Another member commented that pediatric patients should not have similar mortality to adult patients, so pediatric priority must be maintained. Finally, another member mentioned that there needs to be clarification in regards to PSC in terms of how to define sepsis and admission. Rob Mottola | 03/23/2021 I am newly diagnosed PSC, Cirrhosis, Portal Hypertension and UC. This has been a crushing blow to my wife and 4 children. Please support this exceptions proposal and save lives. Thank you. Anonymous | 03/23/2021 I would like to express my strong support for the proposed modifications to the NLRB policy, specifically as it relates to PSC. MELD score alone has repeatedly been shown to underestimate risk of decompensation in PSC, which is grimly reflected in the significantly higher waitlist dropout rate among PSC patients with MELD scores of 37 and above. Any proposal that lowers the bar for PSC patients to be granted exceptions is a positive step toward a more equitable organ distribution system. Region 2 | 03/23/2021 Region 2 sentiment: 11 Strongly Support, 6 Support, 7 Neutral/Abstain, 0 Oppose, 0 Strongly Oppose. Comments: The region is supportive of the proposal. One member expressed concern that for PSC patients switching from ICU to hospitalization alone is insufficient. Many PSC patients are admitted with sepsis and require antibiotics for cholangitis but blood cultures are frequently negative, which prevents them from getting MELD points. Another member inquired as to whether the proposed changes for PSC candidates adequately address the concerns raised by the PSC community. Jan Reicher | 03/23/2021 As a newly diagnosed PSC patient, I strongly support the change in calculating the MELD score. Hopefully with this change it can help save thousands of our lives. Please make this change and save lives. Anonymous | 03/23/2021 Please make it so that PSC patients receive exception points only after they are admitted to the hospital two or more times within a one-year period for a documented blood stream infection or evidence of sepsis including hemodynamic instability requiring vasopressors. I am one of the individuals who have PSC. As you know, the effects of PSC can vary greatly and the disease can progress rapidly. There is a disparity between MELD scores and severity of the disease that cause some to not get the transplant they need. I feel a one year time period for incidences of sepsis is more reasonable. Region 9 | 03/23/2021 Region 9 sentiment: 2 strongly support, 4 support, 1 neutral/abstain, 0 oppose, 0 strongly oppose. During the meeting there were comments voiced during the discussion, submitted online and submitted as comments from the OPTN representatives. The region generally supported this proposal. A member commented that these updates for PSC patients make a lot of sense and that perhaps they should be relaxed further. Terri Doster | 03/23/2021 As a patient I strongly support the proposed changes and clarifications regarding exception points for PSC patients. Quality of life with PSC can be extremely poor and even unbearable while awaiting a transplant. Many of us are sicker than what clinical evaluations show. Sometimes it even affects the will to live, especially knowing that one may never meet the qualifications to receive a desperately needed transplant. Please help us. Al Uluatam | 03/23/2021 I am the father of an 18 year old girl diagnosed, approximately 8 months ago, with Primary Sclerosing Cholangitis (PSC). During this period I have seen her suffer terribly on several different occasions as the liver enzymes built up in her blood-stream due to blockage in her primary bile ducts. Surgical procedures were used each time to open such blockages which I know is, likely, only a temporary remedy. She is likely to become a candidate for a liver transplant in the future. I am not a doctor and cannot comment on MELD scores and related exceptions, but as a parent, I would like the medical profession and various tests/procedures (such as MELD exception scores) to appropriately assess the illness level of PSC patients so that they do not suffer unnecessarily and do have a fair and equitable chance to successfully get a liver transplant. My understanding is that the current system/criteria does not appropriately recognize the serious liver damage suffered by PSC patients and I support the proposed changes which I understand will help remedy the problem. I simply want my daughter to have a fair chance at a successful liver transplant if and when the time comes for it. God bless all. Anonymous | 03/23/2021 I strongly support these updates. As a patient with PSC and someone who has been under observation for cholangiocarcinoma, the transplant process was confusing in terms of how to qualify for exception points. This update would clarify the confusion and help patients better understand what is happening at an already very overwhelming time. I also strongly support the updates to evaluating PSC patients including hospital time. As PSC gets more advanced patients will need more and more treatment, including time in the hospital, which should be reflected in the evaluation for a transplant. Anonymous | 03/23/2021 I am in support of the policy change for exception points for PSC patients. My daughter in law is a transplant recipient and because of recurrent PSC a probable future candidate for a second transplant. Shortly after she was diagnosed with recurrent PSC, she was hospitalized with bacteremia. In that hospitalization and in subsequent appointments, she has been told repeatedly that she will eventually need a transplant and probably wouldn’t qualify for another deceased donor via MELD. This change could help the sickest PSCers. I fear that she may not make it to transplant. That would be a great loss to society because of all the generous volunteer work she does in support of PSC moms and her many creative talents. She is the mother of a five- year-old little boy that will need his mom for many years to come. Pleae give her the gift of life. Thank you. American Society of Transplant Surgeons | 03/23/2021 The American Society of Transplant Surgeons (ASTS) appreciates the OPTN Liver & Intestinal Organ Transplantation Committee’s review of the NLRB process to improve guidance. The policy changes will serve to increase the number of patients with End-Stage Liver Disease (ESLD) who are eligible for an exception via the NLRB. We support the policy proposal overall with the following clarifications: 1. On guidance for growth failure and nutritional insufficiency: These are not adequately accounted for in the current PELD calculation and remain an important risk factor for poor waitlist outcomes. The clarifications remove the age criteria of <1 year to remove disadvantages for infants and clearly define how to calculate z-scores and anthropometric scores for malnutrition in pediatric patients. Overall, the proposed changes in the guidance would provide improved access to exception for a select group of vulnerable pediatric candidates for whom PELD scores do not fully predict wait-list drop out or mortality. 2. On pediatric guidelines: PELD and MELD scores are good discriminators of death without a transplant for many pediatric patients. However, for some patients, complications of liver disease, and not the degree of liver dysfunction, determine the need for liver transplant. Statuses and PELD/MELD scores do not reflect these complications, which have an increased risk of mortality without access to a timely transplant. In general, NLRB reviewers should be more considerate in approving PELD/MELD exceptions for children with portal hypertension complications. We believe that preventable pediatric waitlist mortality should be eliminated based on our overall commitment to this vulnerable population and fairness. 3. On addition of guidance for candidates admitted with ascites requiring IV diuretic therapy or complications of portal hypertension (ascites and GI bleeding): The proposal seeks to define the specifics of these complications more clearly for patients to qualify for PELD exceptions. The ASTS supports clarification efforts to standardize scenarios for children to get additional PELD points and suggest these could be honed further. 4. On Neuroendocrine Tumor (NET) guidance: New literature supports removal of the arbitrary age limit to improve equity and access. 5. On guidance for candidates with PSC: The policy specifies that patients must have had two hospital admissions with bacteremia or septic shock within a year. While this may help stratify this population, it may be helpful to assign a specific score to these higher risk patients. We suggest identification of a standard MELD exception score across the country (MMAT-3 or other). 6. On hilar cholangiocarcinoma standardized exception criteria: We support the proposed criteria which is in line with standard practice and would reduce the number of NLRB applications. However, we suggest the OPTN consider the diagnosis of cholangiocarcinoma to be based on positive cytology only. 7. On metabolic liver disease: This policy clarification opens up rare metabolic diseases to exception appeals but it would be too exhaustive to list every rare metabolic syndrome that exists. Hence, centers that request this appeal must submit literature to support how transplant would ameliorate this rare liver condition. Jennifer Chavez | 03/23/2021 I strongly support the proposed NLRB policy change. For the past two 1/2 years, I have worked with PSC Partners Seeking a Cure. I have come to know and love the thousands of people with primary sclerosing cholangitis (PSC), and their caregivers, family members, and friends. I have witnessed how sick patients with PSC can become while waiting for a liver transplant. In fact, by the time PSC patients reach a high enough MELD/PELD score, they may be too sick to receive a transplant. The NLRB proposed policy change will decrease the inequity created by the MELD/PELD scores for PSC patients waiting for a liver transplant. I support the policy change. Anonymous | 03/23/2021 I am from Australia and wanted to show my support. Region 1 | 03/23/2021 OPTN Liver and Intestinal Organ Transplantation Committee presented by James Markmann, MD, PhD Comments: One member commented they agree with pediatric changes to guidance, and this will be very helpful for pediatric NLRB. Another member commented that patients often have cholangitis and we will need to specify that hospitalization is from cholangitis. Region 1 sentiment: 6 strongly support, 2 support, 3 neutral/abstain, 0 oppose, 0 strongly oppose Jan Reicher | 03/23/2021 I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. Anonymous | 03/23/2021 I strongly support the NLB's recommendation to provide exception points for frequent hospitalizations. This needs to change to save the lives of people with PSC. Ten years following my first liver transplant, PSC came back with a vengeance and I was left facing transplant again. I spent the next 2.5 years with a bag draining my bile and lost so many months recovering from hospitalizations and sepsis. I developed sepsis 6 times in 2.5 years. Despite the many infections, the bag kept me just healthy enough to keep my MELD number far too low for a cadaveric liver transplant. I was only able to get my 2nd transplant and fresh start at health because of a live donor. This was not my doctor's preferred treatment, but we all agreed this was the only path to a transplant. I was so fortunate to beat sepsis every time, but it should not be this way. Please improve the system and help others just like me. Too much of my life was lost due to hospitalizations and recovery and my MELD never reflected the state of my health. Niall McKay | 03/23/2021 I have PSC and I get recurrent and very dangerous infections or cholangitis on my bile ducts. They come on very quickly and I cannot tell how or why I get an infection. I get them every three to six months. When I am infected my Meld score is high. When I am not suffering from an infection my meld score is low. But without intervention (liver transplant) this disease will kill me. The current system seems to be to be like I am playing the slot machines knowing that the house is always going to win. Anonymous | 03/23/2021 I am currently listed with a MELD of 29, and I’m hoping my INR will increase so that my MELD will increase. I was a 33 just 3 weeks ago because my INR was quite high. Unfortunately they had to do vitamin K transfusions to bring it down so I could safely undergo an ERCP. This brought me down to a 27. I’ve just been waiting and waiting for the effects of all the vitamin K wear off. In the mean time, I feel weaker, heavy due to all the retained fluid, and uncomfortable if not in pain all the time. I am mostly stuck to my bed. I barely sleep. My quality of life is bad. My transplant facility does not do living donations and I consider my self one of the lucky PSCers because my MELD is high enough to get me a backup liver, though the MELD calculations do not properly express the actual projected survival rate. Since the changes in 2019, I have increasingly witnessed live donation as the only path to transplant. I hope that more candidates can get the PSC exception points to help them achieve a high enough MELD to actually get a liver through a deceased donor. Chris Sawyer | 03/23/2021 I STRONGLY SUPPORT PSC patients to receive exception points after they are admitted to the hospital two or more times within a one-year period for a documented blood stream infection or evidence of sepsis including hemodynamic instability requiring vasopressors. I have PSC been admitted to the ICU for treatment, pre-sepsis, and I continue to require novel antibiotics each time I have an ERCP or other procedure because I often get infections and we found pseudomonas in my biliary tree. Living with PSC is difficult, especially knowing that there isn’t really any treatment at this time capable of fully arresting the further advancement of the disease in my body, short of transplant. When my time comes to need a liver transplant, it will be because there is no other treatment available. Every opportunity to make transplant more available to those suffering from PSC and who have otherwise run out options should be taken. Please and thank you for your consideration. Chris Sawyer Ryan Leonard | 03/23/2021 I strongly support the proposed changes being considered to the National Liver Review Board Guidance Documents and Policy Clarification. I was diagnosed with PSC last year and have already had several ERCP's for treatment. I already have cirrhosis and living with both Ulcerative Colitis and PSC brings many challenges and uncertainties as you don't realize when the next painful attack might occur. My MELD score has fluctuated quite a bit and as PSC patients know, we can feel very sick with complications but our MELD score might not reflect the severity of our disease. Providing exception points for patients living with PSC will put us in an equitable position along with others who need a transplant. Thank you for your consideration. Anonymous | 03/22/2021 I strongly support the proposed changes and clarifications regarding exception points for PSC patients. PSC patients should receive exception points after they are admitted to the hospital two or more times within a one-year period for a documented blood stream infection or evidence of sepsis including hemodynamic instability requiring vasopressors. I was diagnosed with PSC when I was three years old. At the age of eleven, I was placed on the transplant list. I had severe portal hypertension, recurrent infections, esophageal varices, and adrenal insufficiency. I was at a disadvantage on the list because of my disease and my PELD/MELD score remained low despite being very sick. My doctors lobbied for exception points for me multiple times and were denied every time. We started the process of living donation but out of the dozens of people tested, no one was the right match. I waited for over four years on the transplant list and was finally able to receive a transplant three years ago. At the time of my transplant, my MELD score was still low and I believe the only reason I was able to receive a liver is that my surgeons were able to perform a split-liver transplant to save two lives. It is very possible I could have waited on the list to the point of being too sick for a transplant; this happens all too often in the PSC community. Please consider the proposed changes for exception points. These changes will immensely help PSC patients in need of a liver transplant. Veronica Miller | 03/22/2021 I strongly support the proposal to change the policy for PSC patients to receive exemption points as outlined in your document. The proposed change will more adequately address the significant unmet medical need faced by PSC patients waiting for a liver transplant. I fully support PSC Partners in their advocacy for this change. As the Director of the Forum for Collaborative Research which includes the PSC Forum, I have personally met caregivers and patients suffering while waiting too long for a liver transplant. Thank you for the opportunity to comment Anonymous | 03/22/2021 As someone with PSC please consider not only how the consideration of my disease in conjunction with MELD score would substantially improve my future outlook and prognosis but also what it would mean for my wife, son, family, and friends. Your consideration of a change here would quite literally change the lives of everyone diagnosed but also those silently suffering by our sides. Jennifer Moss | 03/22/2021 I strongly support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My husband was diagnosed with PSC in 2012. He was hospitalized 19 times in 21 months, including a trip to the ICU for sepsis due to a cholangitis infection. Recent statistics show that the dropout rate of PSC transplant candidates with a MELD or PELD score greater than 37 is substantially higher for PSC patients than for other listed liver patients. This data confirms what we as patients and caregivers have long observed. We urge and support you updating the guidelines to give patients with PSC the equitable chance that they deserve. Thank you. Louis Karpinski | 03/22/2021 As a caretaker for a PSC patient, I call on NLRB to finally make this vital policy change. For anyone who has not seen the miserable suffering of the symptomatic PSC patient, it is hard to adequately describe their mental and physical anguish. This policy improvement is the right thing to do! Lisa Bischoff | 03/22/2021 I strongly support the proposed change to include exceptions for PSC patients in regard to the MELD score. My husband is only 37 years old and was diagnosed with PSC one year ago next week... we are expecting our first child this August. It terrifies me to think about our son growing up with a dad who is sick, in the hospital, suffering, in pain, and not knowing how much longer he has... or worse yet, without a dad at all. It is so disheartening that PSC patients are not given consideration and exception when it comes to MELD scores. PSC is not brought upon them by their lifestyle choices, there is no treatment or cure, and they often feel isolated, alone, + scared about their future due to so many unknowns about the disease + its progression. To ask a PSCer to be so close to death to deserve a transplant is inhumane - close to death + deserving of a liver are not always the same thing. My husband and I try not to even think about what his future may hold - begging family + friends to give up their own liver and undergo a serious surgery to try and save him... or waiting until he's so sick that he is quite literally at death's door before he is deemed as worthy as other liver transplant candidates, some of whom have unfortunately made a series of conscious decisions on numerous occasions for years on end to partake in lifestyle choices that brought them to needing a transplant in the first place. If only PSC patients were lucky enough to have the luxury to choose what they could do or not do that would bring them to that point... please, for the sake of my smart, kind, selfless, hardworking, funny, quick-witted, clean freak, cook extraordinaire, golf enthusiast, travel-loving, sick, scared, hurting husband and soon to be dad... please, don't let it come to that for him or anyone else with PSC. Carla Stewart | 03/22/2021 As someone who has a loved one with PSC I support the proposal to allow exception points for PSC. MELD score should not be the only factor used in determining when someone gets a liver transplant. It is an unfair method for patients with PSC. David Barber-Callaghan | 03/22/2021 I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My best friend is on the transplant list for PSC. He follows all of the guidelines and does everything he can to be healthy. At some point in the future he will probably need a transplant due to the natural progression of his disease. Unfortunately, since the current MELD scores don't account for the complexities of PSC, he will probably become too sick while waiting for a transplant. Please support the community of people affected by PSC by changing a scoring system that systematically jepordizies their chance at a transplant. Anonymous | 03/22/2021 To Whom It May Concern, I would ask that you strongly consider this proposal. I was diagnosed with PSC two years ago. Recently, my PSC has shown its ugly head more often. I have a very supportive wife and two kids. I can't sleep at night as I am already worried about what will happen when my liver is ready to call it quits. I cry at the thought that my kids will essentially have to see me standing in line for heaven, waiting for the gates to open when there is a chance for me to get back to being a role model and enjoying family time with my kids but due to some 'red tape rules', I might never get that opportunity. Thank you for your time and consideration. Lisa Pedicone | 03/22/2021 I am a clinical researcher in the field of liver disease as well as the mom of a 20 year old with PSC. My son has suffered for the past 10 years and, like many others diagnosed at a young age, knows that a liver transplant is something likely in his future. The MELD scoring system doesn't account for the severity of disease many PSC patients experience. Poor quality of life due to constant, uncontrollable pruritus, recurrent cholangitis attacks, sepsis, and frequent ERCPs are part of everyday life for many with PSC. Most of the above consequences do not result in ICU stays but do result in frequent hospital admissions especially as the disease progresses. PSC is a rare disease and the consequences are unknown to the non-PSC world. For those of us impacted by this disease without a cure, the consequences are real and devastating for many. The current proposal to modify the granting of exception points for PSC will not overwhelm the current allocation system with PSC patients but will give those with this autoimmune disease a better chance to live life to the fullest. Thank you for considering this change. Carly Barber-Callaghan | 03/22/2021 My friend is on the transplant list for PSC. He works so hard to live a healthy lifestyle and to prioritize wellness, however his disease progression is out of his control. He will likely need a transplant in the future. Because of the current standards, he is much more likely to become too sick or die waiting to be transplanted than any other listed liver patients. We are fearful of losing him to a disease that can be treated by transplant because the MELD scores do not take into consideration the complexities of the PSC disease. Please support my friend and the community of people affected by PSC. American Society of Transplantation | 03/22/2021 The American Society of Transplantation is grateful for the continued review of the NLRB guidance criteria and supports the proposal as written with no further comment. Anonymous | 03/22/2021 We write in support of the recent initiative regarding allocation of organs for liver transplant. For many years we watched our college-aged daughter battle the ravages of PSC knowing only too well that the system in place would prevent her from ever getting a much-needed liver transplant. Finally in desperation we moved forward through the terrifying ordeal of a 19-hour live donor transplant between our two children. No family should have to go through this. The current system failed us. The proposed changes will go a long way toward resolving this problem. Nick Puckett | 03/22/2021 I strongly support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. This would be a far more effective method of determining the priority level of a candidate vs. the status quo. Many people with PSC end up passing away due to the fact that the current system overlooks many of their symptoms and/or complications. I was diagnosed with PSC in 2010. I have been relatively asymptomatic for the past eleven years, however, the periods that I have suffered setbacks from this disease have been rapid and brutal. I have been hospitalized once with pancreatitus due to PSC, once with sepsis via a cholangitis attack, and a handful of other times with less sever bouts of cholangitis. Please review all of these comments and know that some people with PSC don't make it past their 2nd or 3rd year after diagnosis. This is a very difficult disease to track and nailing down a long term prognosis for a patient can be even more difficult, but that doesn't mean we shouldn't try. We have to ask ourselves every opportunity we have, "What can we do to save more lives?" This motion is absolutely a step in the right direction. Thank you for your time and consideration. Anonymous | 03/22/2021 I worry every day for my dear family member who has PSC. With the MELD system as it is, I fear she will have to get very ill before, hopefully, qualifying for a donor liver. Please adjust the system to be more fair to those with PSC! Robert Stephens | 03/22/2021 As the father of a PSC patient I am very concerned that she may not be eligible for a liver transplant in the future should she need it. I have witnessed first hand the misery this disease has already caused my daughter - even in its early stages. I believe she and other PSC sufferers should be eligible for transplants as their disease becomes increasingly unbearable. Madison Stephens | 03/22/2021 I strongly support the NLRB proposed changes to guidance for candidates with PSC/SCC. The experience of my recurrent and debilitating PSC symptoms is made much worse by the daily psychological burden of knowing there are limited treatment options. Upon receiving my frightening diagnosis, I was informed that I will be unlikely to benefit from the MELD score system as it is currently structured if I should one day need a life-saving transplant. The proposed changes are a step in the right direction. Robert Collins | 03/22/2021 Dear Review Board- It is of critical importance that you begin the process of reversing the decision to stop giving PSC patients exception points. Exception points for PSC patients MUST be restored. In 2019 my 20 year old son. Had received 18 bandings for bleeding varices over 4 different procedures. After violently vomiting blood and admitted emergently to the hospital He was both listed and given exception points to a MELD 25. We were instructed by his doctors that under the old system he would have been eligible to not only retain this number but ask for an additional 3 points with in 90 days. Thus almost ensuring transplant within the next 3 to 6 months. Then the change came! My son not only did not get future exception points but all exception were taken away and he was now a MELD 14. Desperate and well aware what this meant we aggressively pursued living donor. If it was not for this avenue my son would have died for sure. After my wife risked her life to donate half her liver to my son this is what his liver looked like. Please see the attached picture. This is his liver after transplant. He clearly was much sicker than a MELD 14 but this was the system you created. It must be fixed. In an effort to be fair, all you did was make it horribly unfair. Yes is a 65 year old alcoholic more likely to be transplanted today, yes. Is it at the sake of a 20 year old who never drank in his life and by no fault of his own is now dying. The answer is YES. You are trying to make it so the patient closest to death gets the liver first, but you are not taking into account who is most likely to have successful outcomes with the organ as well. only now after PSC patients are dying more, you are willing to fix this. I could have told you this a year and half ago this was going to happen. Being the closest to death and being the most deserving are not the same. With a greater than 20% relapse rate for alcoholic liver transplants, you MUST consider exception points for PSC, this is the system that then allows doctors in the field with peer review to make the "RIGHT" decision. This is why PSCers were getting so much exceptions before. Because they were otherwise people who didn't eat or drink themselves into liver failure, and the doctors knew the exception system could allow them to move more deserving patients in front of less deserving and also produce better outcomes for their respective centers as a result. I recognize most of you reading this are Medical Doctors. Having lived through this process, I would encourage you to stop making decisions on primarily statistical data, and start talking to your staff. The transplant nurses in any transplant wing in America will tell you exactly who the patients are who will fail to comply, and who will be successful. Personally, If your whole board was made up of transplant nurses and they decided who received organs it would be far better system then your current system. Please I beg you for future patients, Make a system that rewards the deserving, not just one that rewards the most influential. Global Liver Institute | 03/22/2021 Under the proposed changes to the National Liver Review Board Guidance documents and Policy Clarification, candidates with cholangiocarcinoma (CCA) would have to meet additional diagnostic criteria including a hilar mass of 3cm or less in radial mass to qualify for automatic exception points. We understand the need to align policy with current guidelines, but this proposed update to the NLRB Guidance for CCA places additional restraints on an already exacerbated situation in the individual already living with cancer. In addition, liver patients with primary sclerosing cholangitis are at a greater risk for CCA. For candidates with cholangiocarcinoma, liver transplantation in conjunction with chemotherapy is the only choice at a better quality of life, so why create additional barriers for candidates to overcome by adding an additional requirement for automatic exception points? According to the Cholangiocarcinoma Foundation, CCA patients undergo two obstacles when seeking a transplant, one, very few livers are given to patients with CCA because transplant centers prefer to use donor livers for curable diseases, and secondly, not many transplant centers accept patients with CCA, thus leaving patients with CCA vulnerable to complications the longer the wait for transplantation. There is an urgent need for better guidance documents and clear policy guidelines for equal distribution of deceased and living liver donations for patients with liver cancers and liver diseases. The second part of the proposed NLRB Guidance Documents and Policy Clarification, would add guidelines for primary and secondary sclerosing cholangitis, neuroendocrine, and pediatric exemptions. Research studies have shown that PSC transplant candidates receive lower MELD Scores due to lack of medical urgency or progressing diagnosis not documented by the MELD Score. As stated in the American Association for the Study of Livers Diseases Evaluation for Liver Transplantation Guidelines, liver transplantation is an effective treatment for patients with PSC and decompensated liver diseases when there is a recurrence of cholangitis or sepsis. Patients with PSC become gravely ill before being granted higher MELD scores. For PSC candidates with higher than 37 MELD or PELD Scores, large waitlist drop-offs can be attributed to the MELD Scores inability to include the complexities and mortality of candidates with PSC and other additional progressive liver diseases. The proposed guidance document updates are suggesting adding PSC, SSC, Nets and Pediatric Exemptions as a solution to increasing equity in transplantation, but the liver community sees a continuous imperfect solution to a broken system filled with health inequities. The NLRB members suggest the proposal of removing the age threshold for neuroendocrine tumors which is currently at the age of 60. Age limitations in organ allocation is a misconception that older organ donors and older persons needing organ transplants should not qualify for they are at higher risk for transplant complication. This age limitation should not keep a potential recipient from receiving viable organs from an older donor, or an older candidate from potential transplantation. A full work-up assessment should be done to assess the pre-transplant and post-transplant survival waitlist, and new guidelines set for NETS and other liver cancers that factor in age thresholds. For pediatric exemptions, the PELD Score has its disadvantages, because children with certain PELD Scores receive longer waitlist times and growth failures methods don’t take into account the higher waitlist mortality rates for children. A solution to this problem, splitting livers to be donated between children, allowing more children to be transplanted in adequate timing. An additional solution is altruistic directed and non-directed to increase national access to the donor pool for both children and adults. As reported by Dr. Vatche G. Agopian, the PELD and MELD Scores introduction as measures to transplantation, children received a lower rate of transplantation at 41% compared to 48% who received deceased donors prior to the introduction of this system. The proposed updates create a competition between disease states, dividing the liver community. We propose the OPOs take into consideration the use of “true science” by collaborative reform, including patient voices, donors, the scientific community, and discussions from the American Association for the Study of Liver Diseases Liver Meetings to design a new equitable system that addresses health disparities of vulnerable populations and the complexities of disease states for liver transplantation and the quality of life before and after transplantation. Finally, African-Americans face the greatest hurdles from getting on the waitlist, to finding a match, to becoming a donor. Patients of color who need an organ have more adverse experiences within an organ donation system deeply rooted in inequity. 7 Previous studies suggest implicit bias and preconceptions disproportionately affect people of color. 8 In response to this crisis, we need urgent congressional oversight and administration action! Strategies must address racial obstacles to getting on the waitlist, increasing medically suitable organs available for patients of color, and improving the likelihood of potential donors of color to be referred. In some cases, OPOs have not shown up to talk to donor families of color, failed to get proper authorization, nor found a correct donor/organ match. As stated by the 2021 UNOS TMF speaker, Amy Hunter, ph.D, and CEO of Lotus Roots Consulting LLC, “We must undo, dismantle and interrupt the patterns within our system and communities that have led to disparities in transplantation.” Attachment Jerome Schofferman | 03/22/2021 I strongly support the NLRB proposed changes and clarifications. I am a PSC patient and transplant recipient as well as being a physician. I had multiple episodes of infectious cholangitis that required frequent hospitalizations followed by antibiotics at home via PIC line. Episodes of infection became more frequent and eventually I was in ICU with bacteremia and sepsis. Despite clinical disease progression, my MELD score remaind low. As a result, I was denied exception points several times until after my episode of sepsis with hypotension requiring pressors. It became clear that I was at high risk for death due to infection. At that point I did received exception points, subsequently underwent transplant and have been doing well. The new NLRB guidance is extremely important to help PSC patients such as myself and could save many lives. Anonymous | 03/22/2021 As a patient I support this change to provide exception points to PSC patients: I have had PSC since 2009, it has been a hard to fight disease with no FDA standard of care or practice. And no real medicines that work to mitigate the effects of the disease. As time moves on the immune system continues its attack on the liver causing fibrosis and eventually the liver no longer functions. I would appreciate your support of the proposed change for PSC patients. thank you. " PSC patients receive exception points only after they are admitted to the hospital two or more times within a one-year period for a documented blood stream infection or evidence of sepsis including hemodynamic instability requiring vasopressors." Eric Vivio | 03/22/2021 My sister Laura Vivio-Karle has PSC and is in need of exception points on the donor list for her liver. She is married with two young daughters. Her liver is getting really bad. We need your help to get her a liver transplant. She is a physicians assistant, a professional career but she cannot work very much these days. Due to her PSC she has lots of pain, multiple scopes and also infections. Unfortunately, there are people with higher meld scores due to their drinking and are on their second lover transplants. DRINKING which is a self-induced condition. That is ridiculous! People with PCS are lower on the donor list than people with alcohol induced liver problems. PSC is something that is UNPREVENTABLE! I'm asking you to please get the PSC patients their exception points so they can live a better life! I love my sister very much and she deserves your help! Anonymous | 03/22/2021 I strongly support exception point eligibility for PSC patients awaiting transplant. The concern is that the MELD score for those with PSC does not adequately reflect the severity of the person’s disease. My daughter who had PSC suffered with cholangitis, sepsis, HE, constant unbearable itching, autonomic dysfunction, nighttime sweats and migraines. Her quality of life was poor, but she made the most of it. The doctors told her she would not be able to use a living donor (she had a volunteer), that she would need a deceased donor. With treatment her MELD would constantly jump from 15 up to around 40, then back down. This frequent fluctuation had her going to the top of the list, way down on the list, back up, etc. She was finally called in 2 times, with each being a provisional liver (deceased donor age 70+). Neither proved to be viable. Our daughter’s condition continued to worsen and she became too sick for transplant. A sad by-product of our daughter’s progression of PSC resulted in a host of other medical issues, and organs being damaged. I firmly believe that the current MELD system contributed to her demise. Christopher Porter | 03/22/2021 As a transplant recipient who knows what it's like to be on a transplant list, I support better access for patients living with PSC. NATCO | 03/22/2021 NATCO commends the Liver and Intestinal Committee for working to improve guidance with the intent to ensure those patients whose score does not reflect their need for transplant are not overlooked by the current system. We believe the proposed addition will provide clarity for the transplant centers and Liver Review Board and do not foresee any risks with the proposed change other than a slight increase in waitlist. Anonymous | 03/22/2021 This is a much needed update to the policy. Time to make these changes. PSC transplant candidates should receive exception points if they are admitted to the hospital two or more times within the last year! I have seen how PSC can afffect patients much differently than other liver diseases and the allocation of exception points, or lack of it, has not been fair. Anonymous | 03/22/2021 I strongly support the allowance of exception points to be added to the MELD scores for PSC patients. The MELD system is extremely unfair to PSC patients, and despite having no control of their progressive disease, they are penalized among all transplant candidates. My nephew was diagnosed as a young child and we hope that if he needs to be considered for transplant at some point in his life that he will be able to get one without risk that he will be made to wait until he is too sick to be considered. Daniel McNamara | 03/22/2021 I was diagnosed with PSC at 24, was listed for transplant at 32. I was told I would most likely die if I waited for a liver transplant using the meld score. My cousin volunteered to donate half his liver to save my life. I'm now 37 and as healthy as I can be given my diagnosis. If the MELD scores were revised when I needed a liver, it's possible my cousin wouldn't have needed to volunteer. How many friends and relatives put their lives on the line for someone with PSC when they didn't have to because the MELD score was biased towards PSC patients? The current system doesn't work for PSC patients and their families. The amount of sacrifices that patients and families have made because of that fact should be on the minds of everyone who is going to make this decision. Liver transplant isn't a cure but is currently the only treatment for the eventual outcome of liver failure due to PSC. This disease punishes people enough, no need for further medical bureaucracy to get in the way or to leave them to die. PSC is a serious liver disease and unusual and PSC patients deserve to be fairly represented when in need of a liver. It's a rare disease that needs more attention and is always overlooked by the medical community. Patients suffer because of that so please fix whatever you can now to save more lives now. Anonymous | 03/22/2021 Our little 7 year old grandson is a PSC Patient. If he ever will need to be considered for a transplant, we pray he will be able to get it without risk that he will have to wait until he is too sick to be considered. Thank you. Amy Forsyth-Stephens | 03/22/2021 I am in favor of adding persons with PSC to the list of candidates for liver transplant. My daughter, age 31, is a PSC patient who will likely need a liver transplant to live past middle age. I hope that she will be a candidate when the time comes. Anonymous | 03/22/2021 I strongly support the allowance of exception points to be added to the MELD scores for PSC patients. The MELD system is extremely unfair to PSC patients, and despite having no control of their progressive disease, they are penalized among all transplant candidates. My nephew was diagnosed as a young child and we hope that if he needs to be considered for transplant at some point in his life that he will be able to get one without risk that he will be made to wait until he is too sick to be considered. Anonymous | 03/22/2021 My sister has PSC and she will likely require a transplant at some point in her disease journey. With the current deceased donor allocation specifications, I worry that I will have to watch my sister get severely ill and maybe even die, because of the MELD score required for someone with PSC to qualify for a liver. Please help save my sister by adjusting the MELD system to more fairly capture the severity of her illness and her transplant needs. Thank you for allowing me to make a public comment. Anonymous | 03/22/2021 I strongly support the allowance of exception points to be added to the MELD scores for PSC patients. The MELD system is extremely unfair to PSC patients, and despite having no control of their progressive disease, they are penalized among all transplant candidates. My son was diagnosed as a child and we hope that if he needs to be considered for transplant at some point in his life that he will be able to get one without risk that he will be made to wait until he is too sick to be considered. Anonymous | 03/22/2021 As a liver transplant candidate I support this change. Two close friends and far too many acquaintances have passed away due to being unable to receive a transplant in time. Had this change been in place, they would have been prioritized differently and possibly still with us. Enacting this change also reduces undue suffering by expediting transplant qualification. Thank you for the opportunity to share my opinion. Kelsie Page | 03/22/2021 I am writing to support this proposed policy changes around exception point eligibility for PSC/SSC. I am the parent of a young adult with PSC. It is unfortunate that PSC patients MELD score does not take into consideration how ill a patient with PSC may be. Thank you for your consideration Monica Crowley | 03/22/2021 My husband John was diagnosed with PSC at age 33 and received a living donor transplant at age 46 after years of recurring hospitalizations for infections. When he was transplanted his MELD hovered around 14 - which did not reflect the wasting, recurring infections, and his risk of bleeding to death from varices in his stomach and esophagus. Luckily he was able to receive a LDLT - the photos of his liver after the transplant as many commenters noted - shocked the transplant team because there was such extensive damage that was not reflected by his low MELD - and the surgeons said he would not have lasted many more months without the transplant - regardless of what the MELD said. My husband is and has been one of the most compliant, proactive, participatory patients that most of his doctors have ever worked with. He is also a father and a small business owner with over 30 employees who also depend on him. And most PSC patients I know are like this - active in the face of their struggles, caring, participating fully in caring for others - and this change can give them a fighting chance at survival. Joanne Hatchett | 03/22/2021 Thank you for proposing these positive changes. PSC patients usually hear about the need for transplant when they are told about their diagnosis. In the 16 years since my son was diagnosed, we have known too many PSC patients who have either died or been told they were too sick for transplant. PSC patients and families live with the fear that suddenly or over a long time, they will live with the symptoms of end-stage liver disease, and the cycle of critical illness, hospitalization with treatment, followed by a drop in their MELD score. The cycle repeats along with progressive symptoms, increasing debility, and the risk of either sudden death, or progressive decline and the possibility being told they are too sick to receive a transplant. The proposed guidance changes bring hope to change this cycle. My request is for the NLRB to develop specific annual monitoring criteria to evaluate PSC outcomes following these changes. I support the proposed PSC guidance changes. Anonymous | 03/21/2021 I am the mother of an adult PSC patient. I’ve spent years at PSC Partners volunteering for the PSC community - Wonderful years sprinkled with moments of anguish and helplessness. I’ve dreaded hearing the words that come in different shades and from so many: “I’m jaundiced; I’m in endless pain; I can’t get out of bed; the exhaustion is impossible to shake off; I can’t focus and think straight; I want to roll in the snow to numb that overwhelming itch; my MELD score is 13.” I’ve lost dozens of friends to PSC, men and women who were filled with plans for the future, mothers and fathers wanting to live to see their children grow up. One of them was 37, a leader in the PSC community, a beautiful young woman I got very close to through the years. I flew across the country so I could give her a last hug. She wasn’t giving up: In her final days, she found strength in herself to whisper the tune and the words to, “What a Beautiful World,” and in her extreme weakness made one last effort to plan for a PSC Partners fundraiser for research that could save others. How could the system have taken her? Why couldn’t she have the transplant that could have kept her among us? The MELD score never reflected the seriousness of her disease, and she didn’t meet the hospitalization and medical criteria required for a transplant. She died full of life and beauty, and she is still very alive in so many of us. Thank you for taking the first very important steps to save the lives of many in our beautiful, strong, and unified PSC family. Anonymous | 03/21/2021 As a 20 year patient of PSC I can first hand attest to the level of sickness one can experience even with relatively low meld scores. I am a post-transplant patient. I was lucky in that I was transplanted with at a MELD score of 21. The first time, I was noticing symptoms I was already at MELD SCORE of 18. At that meld score I experience extreme exhaustion, jaundice, and weekly paracentesis procedures. By the time I reached a MELD score of 21 I could barely get out of bed for days at a time. I had multiple hospitalizations due to exhaustion, dehydration, and nausea at that time. If you asked me if I wanted to take the risk of transplantation, I would have emphatically said yes! And all this is at a score of 21! I can't imagine how sick people have to get to get to a score like 36! 'How sick do I have to get?' I thought to myself while basically giving up hope that I'd ever receive a liver. Exception points for multiple hospitalization trips in a year's time would give us patients a life line that we desperately need. Even now, after being lucky to receive a transplant, I wonder how sick I'll have to eventually get if I should ever need another transplant. Please allow exception points for PSC patients who are suffering. Larry Stahl | 03/21/2021 My name is Larry Stahl. I'm a 70-yr. old liver transplant patient with a previous diagnosis of end-stage cirrhosis due to PSC. I have written about exception points in MELD scores for PSC and PBC patients on 2 other occasions. I am again writing on that subject, first of all to thank UNOS and NLRB for their excellent work and attention to this topic that was very personal to me. I was transplanted with a non-living donor liver in Jan. 2018. Prior to that I had a MELD score of 11 and had completed transplant evaluation at both Univ. Of Colorado and at Piedmont Hosp. in Atlanta GA. My MELD score at the time was 10-11 and I had trouble getting listed at age 66. Fortunately, I developed a second episode of acute cholangitis with sepsis and shock. As I can recall, my BP in the ER was 55/25. I was stabilized in the ER and had a blood culture which was eventually positive for klebsiella. I was put in a step-down unit instead of ICU bed and treated with IV antibiotics. I improved over 5 days and was discharged. I was actually cared for in a community hospital 5 hrs away from my hepatologist. When my hepatologist found out about my sepsis admission, he wrote a letter requesting exception points for me and I eventually received 4 exception points which brought me to a MELD of 15. At the time, this allowed me to be listed at Piedmont in Atlanta GA. I knew that I had end-stage cirrhosis with ascites, varices that required banding, and hepatic encephalopathy serious enough to require my taking disability from my Urgent Care physician job. Weeks after being listed, I received my liver and did well post-op. I am now 3 years post-transplant, very grateful, and doing very well but have not returned to medicine. I support the proposed changes to exception point policy, even though I understand that these changes would be inadequate at this time to get me the transplanted liver that I needed then. However, I believe this is a robust step in the right direction. I believe in my reading that there is physician-directed movement toward quantifying severity of decompensation . This might make exception points unnecessary. Thank you for your attention to this, and so many letters from my peers. Thank you all for your heroic service to us, especially through this most difficult pandemic year. Maya | 03/21/2021 I strongly support this policy clarification and shift so that PSC patients are more fairly represented and treated. I've had to see my sister go through her PSC experience and unravel the world of liver transplants, fearing that one day she may need one. These little changes have huge implications on the lives of people on the transplant wait list. Amy Hollman | 03/21/2021 I am the parent of a vibrant 18 year old who was recently diagnosed with PSC and experienced significant disease progression over a two-month period. I am writing in support of the proposal to grant MELD exception points to PSC patients who are admitted to the hospital two or more times in a one-year period with a blood infection or evidence of sepsis with hemodynamic instability requiring vasopressors. Since my child's diagnosis, I have spent a significant amount of time educating myself about PSC, and I have been terribly disheartened to learn of the significant discrepancy that can occur between a patient's MELD score and disease severity. Our child's hepatologist, an expert in PSC, recently told us that he and his colleagues see better outcomes with deceased donor transplants than with living transplants, which further convinces me of the importance of taking steps to insure that PSC patients have a fair shot at obtaining a deceased liver before it is too late. Thank you for your consideration. Stacy Goetz | 03/21/2021 Please provide the amended policy for exception points for PSC patients. At the age of 45, my life consists of fevers, feeling absolutely terrible, and constant, and I mean constant, itching. My quality of life has very little enjoyment or value due to the transplant criteria associated with PSC. I could overeat or drink heavily and qualify for a transplant but cannot when handed a debilitating disease that is outside of my control. Jan Maly | 03/21/2021 I strongly support the proposed policy changes regarding exception point eligibility for PSC patients. I am the parent of a 16 year old daughter who was diagnosed with PSC at the age of 11. She is currently doing well, for which I am thankful; but, I know she may one day need a liver transplant. I also know that the MELD score does not accurately reflect how sick PSC patients truly are. If current standards remain unchanged, I worry about some day losing her to this disease. Please support all those affected by PSC. Anonymous | 03/21/2021 In 2019, I learned my teenage daughter has PSC. I quickly researched and joined the PSC Partners team. This is a terrifying disease. In 2020, I learned another of my daughters has PSC as well. Please don’t make my young PSC girls wait until it is too late and they become too unhealthy to get a chance of a liver transplant. Please allow PSC transplant candidates to receive exception points if they are admitted to the hospital two or more times within the last year Christine Petersen | 03/21/2021 As a primary sclerosing cholangitis patient and a physician, I support the proposal to change the regulations regarding exception points in PSC. We often have disease severity out of proportion to our MELD scores and consequently may not qualify for a liver transplant, which is the only proven disease-modifying treatment for our condition. I strive every day to help my patients live the best life possible, and hope to do so for decades to come. I hope that if my disease ever progresses to the point of needing a liver transplant, I will receive a fair consideration for such a precious gift of life. Thank you all for your consideration. Johanna Dodge | 03/21/2021 I was diagnosed with PSC at the age of 31. I’ve watched PSC friends fight the uphill battle of being listed and de-listed due to the current guidelines. As a family, we have been able to remain positive knowing that technologies and procedures change over time. Approving this change is a manifestation of moving forward in regard to liver transplants for us. It is an easy change, but one that will have a huge impact on current PSC patients now and in the future. I support this move and hope you will move forward with the new guidelines. Liver disease isn’t a one size fits all disease and neither should the treatment options be. Thank you for your consideration. Anonymous | 03/21/2021 As a transplant recipient with PSC, I am in favor of updating/upgrading the manner in which transplants are decided upon. People with PSC seem to be penalized because their MELD scores are too low. The current manner of figuring who gets a new liver just doesn't work fairly if folks with PSC cannot get access to life-saving livers. Kelsie Page | 03/21/2021 I am writing to support this proposed policy changes around exception point eligibility for PSC/SSC. I am the parent of a young adult with PSC. It is unfortunate that PSC patients MELD score does not take into consideration how ill a patient with PSC may be. Thank you for your consideration Heather Whiteman | 03/21/2021 I was diagnosed with PSC in 2001 and received a living donor transplant in 2017 after 4 years of severe illness and disability which included 8 hospitalizations, acute on chronic liver failure, bile duct perforation during an ERCP which led to peritonitis, multiple intraabdominal abscesses, home PICC line for months with IV antibiotics and internal/external PTC drains for 4.5 years with tube changes every 60-90 days in interventional radiology and bile drainage bags. I was disabled (from my job as a nurse practitioner) and could not work, lost around 75 pounds and was wasting away, also suffering from extreme itching, fatigue, pain and insomnia. I was told my only chance of transplant was living donor with a MELD of 15-17 at the time. I received a living donor transplant and 7 months later was back to work and have a vastly improved health and quality of life. I implore you approve exception points for PSC patients with 2 hospitalizations for sepsis as I was severely in need of a transplant with a low MELD which is often the case in PSC. Richard Seifert | 03/21/2021 I'm strong support the proposed changes for PSC patients receiving exception points after two or more hospital stay with in 12 months. As a PSC patients myself I not only know the that this change would affect me positively , but have seen negative affects the current policy has had on my fellow PSC patients. I will use my own story as an example. I have been diagnosed with PSC for 17 years. I have significant fatigue and get cholangitis several times a year. I've learned throughout my battle with PSC when a hospital stay is needed. I know not to wait long enough to require an ICU stay, but unable to control a blood stream infection from home. Some times these infections come on within hours of feel stable. We try and do the best we can by staying out of the ICU, but this hurts use by staying lower on the list. I feel my quality of life and that of my family has suffered from the the current policy. Thank you for considering this policy change. Rick Seifert Shelly Morrison | 03/21/2021 As a PSC patient. I am sicker then my blood work shows. I would strongly support this change. Anonymous | 03/21/2021 Statistics show that patients with PSC and advanced stage liver failure are not sufficiently accounted for in the current guidelines. The updated changes are one crucial step, among many others needed, to begin to properly account for the complexities of this disease in relation to organ availability. Sandra Burke | 03/21/2021 I am writing to support the NLRB guidance and policy clarification related to patients diagnosed with Primary Sclerosing Cholangitis. .My son, Philip Burke was diagnosed with PSC when he was 19 years old and was listed for transplant several times during his illness. During those years his MELD score varied considerably and at times meant that his name was removed from the list. Later he was again added to the transplant list and remained there during the last 2 years of his life (he died at the age of 40, having finally received a transplant when his MELD had risen to almost 40 - - after several years of dealing with repeated hospitalizations and infections). His quality of life was diminished significantly in those last few years, and the MELD score did not reflect that adequately. I fully support the modification of the NLRB guidance and policy related to transplant guidelines for those with PSC. If these changes had been in effect during Philip's lifetime, he would have qualified for a transplant sooner, and his chances for survival would have been greatly improved. Anonymous | 03/21/2021 Please allow exception points for PSC patients again. Unlike alcohol related liver failure, PCS patients made no lifestyle choices that increased their risk for their disease. Without exception points, The MELD scoring system only based laboratory and blood-work results, which do not accurately reflect a PSC patients condition or quality of life. Maureen Budjinski | 03/20/2021 In 1999 just before college graduation my healthy son went for a routine physical where the blood work caused a callback. After testing he was diagnosed with PSC then later with Crohn’s. He did everything right but then this. He started a new career but was always following up with doctors appointments. Finally starting to feel ill in 2008 both diseases continued getting worse and was on different medications. By 2012 he was sick enough to be listed for transplant. He started getting complication after complication along with a total colectomy in 2013. All this time his Meld Score rose but not enough. He then developed severe osteoporosis which resulted in kyphosis. He started being hospitalized more and more often with pneumonia and collapse lung. He would not have been able to be intubated at this point. He passed away in June 2015. I feel if he received extra exemption points for these problem he may have had the transplant shortly after being listed. Thank you for listening to this mom who misses her son so deeply. I pray for all the others and care so much for them. Anonymous | 03/20/2021 Thank you for the opportunity to comment. I am writing to support the proposed policy changes around exception point eligibility for PSC/SSC. Meld scores are only one part of the story. In early March 2020, my 60 y/o husband was in great health. Following a gallstone attack that induced biliary blockage, triggered severe necrotizing pancreatitis and sepsis, my husband was diagnosed with Secondary Sclerosing Cholangitis with major damage to his liver biliary. His only option to recover his health is a transplant. Yet his natural MELD score hovers around 14. Due to his ongoing hospitalizations for bacteremia and sepsis, (7 in 5 months) and his increased antibiotic resistance, he was just recently granted the full exception points, but we worry it might be too late. Too sick to travel to another region with lower median transplant scores, we hope that in high-score Region one, a liver will be found before infection overwhelms his already weakened body. Although acknowledging the need to create equity around precious donor allocation, sole reliance on one set of criteria disadvantages patients who are critically ill with fact sets that also support prioritization. I appreciate the opportunity to share our story. Mike Hansen | 03/20/2021 As a patient, I support this change. Fred Sabernick | 03/20/2021 I am both a transplant recipient and post-transplant recurrent PSC patient who will soon be re-listed for a 2nd liver. The only reason I am not listed today is that I could not complete the pre-list testing due to frequent cholangitis infections, most recently last week. In 2021 alone I have been hospitalized on four separate occasions for biliary infections despite the placement of an external drain in January. When I am finally re-listed it will likely be with a MELD between 15-20 despite the severe reduction in quality of life. I am strongly in favor of the proposed change to exception point qualification for PSC patients. I had intended to write more but honestly I am just too tired. Thank you. Anonymous | 03/20/2021 I am writing to support this proposed policy changes around exception point eligibility for PSC/SSC. I am the parent of a young adult with PSC. It is abundantly clear to the PSC patient community that the MELD score does not capture how ill a patient with PSC may be, and we watch helplessly time and time again as our PSC friends languish on the wait list with moderate scores, in and out of the hospital, with highly variable blood work, hence variable MELD scores, only to reach a point that they become too sick to survive transplantation. "Dropping off the waitlist" means a preventable death. The reality of this possible eventuality of not being healthy enough to survive transplant weighs very heavily during the long slow seemingly inevitable decline towards transplant all the while knowing that survivability would be improved if the PSCer could be transplanted before reaching that point. We are realistic that this policy change is a very small step in addressing the real underlying problems of the MELD score not reflecting how sick a PSCer is and the inadequate supply of donor organs, and appreciate the care that the system expends to ensure equity in allocation across all indications for transplantation. I will watch to see how this policy change affects relative outcomes between candidates with PSC/SSC vs all candidates at those high MELD score levels, and request that the NLRB make available a similar analysis as was presented in the education materials regarding this policy change. Thank you for the opportunity to express support for this policy change. Anonymous | 03/20/2021 Eighteen months ago and at the age of 9 years old, my son was diagnosed with Primary Sclerosing Cholangitis. While we are very fortunate that he is nowhere near needing a transplant at this time, my heart aches to think about having to watch him deteriorate to a point that he is so incredibly sick before we can fix him. I respectfully request a policy update to accommodate PSC patients. Anonymous | 03/20/2021 I am writing to support the MELD exception points for patients with PSC (Primary Sclerosing Cholangitis) who have been hospitalized 2 or more times in 1 year due to blood infection. As we know, a PSC patient’s MELD does not always accurately represent the true level of illness in a patient. In addition to level of illness, many PSC patients have “hidden disease” such as cholangiocarcinoma and much more severe illness then blood work and scans show. My husband is post transplant and was transplanted during a time when MELD exceptions were allowed for multiple sepsis hospitalizations in one year. Not only was his liver in much poorer shape then believed with blood work and scans, his explanted liver had high grade dysplasia. Had he not be granted these exception points, it is very likely that he would have died either from the infections, worsening liver function, or from the development of cancer from the dysplastic cells. I believe that these exception points are crucial to insuring our PSC patients can have this unfortunately unique portion of their illness (sepsis, cholangitis) recognized as a contributor to liver transplantation need. I am so thankful that my husband received his Gift of Life and thank all who made this possible. Thank you. Anonymous | 03/19/2021 As a 24 year old newly diagnosed with PSC, it was a shock to hear from my doctor's that there was a good chance I would die before receiving a deceased donor liver and that I should strongly consider searching for a live donor. After 5 years of monitoring (and 3 bouts of pancreatitis from ERCPs), it became time to get listed for transplant. I was fortunate enough to have my younger brother be a match and be willing to donate. At the time, two different hospitals confirmed it was time to get transplanted. My MELD score was 7. To be a young person knowing that I would have to get seriously and repeatedly ill just to have a chance at receiving a deceased donor organ, I thought my life was over. I wouldn't be able to keep a steady job and would have to give up my life to hope for that all important phone call. I'm one of the lucky ones who was able to receive a live donor organ, but I'm also aware that my disease could come back and live donor might not be an option that time. Please recognize the discrepancy between severity of illness in PSC patients and MELD score and pass proposals to correct it. Our lives depend on it. Anonymous | 03/19/2021 I strongly support the proposed update to the NLRB guidance documents and the policy clarification.. My daughter is a two-time liver recipient due to PSC and recurrent PSC. After five months on the waiting list for her first liver transplant (at age 15) she had a life-threatening variceal hemorrhage that put her in a coma for three days. At that point, her transplant doctor told us that she wouldn't live to see the top of the transplant list and that we had to line up a living donor. Under the regional review board exception system, her doctors appealed at least five times for exception points, to no avail. I, two of my sons, and my nephew went through the exhaustive living donor evaluation process. Though my daughter received a living donor (domino) transplant, her PSC returned two years later and she grew increasing ill once again. For four years, her itching was intractable and she tried many medications and treatments with only limited success. Between her first and second transplants, she was hospitalized about 25 times with serious infections - but our capable hospitals did not need to put her in the ICU to treat them. She had PICC lines placed 10 times, until her veins couldn't take it anymore, and then she had a port placed, which stayed in place before and after transplant, five years in all. When she needed to be listed the second time, we listed her in two regions, still under the regional review board system. This time, BOTH regional boards recognized that she was gravely ill, despite a MELD score of just 12, and both awarded her a MELD 30 score. It still took another five months at MELD 30 before she received a deceased donor transplant. She was quickly deteriorating, dropping weight rapidly, losing strength, etc., at age 21. Under the 2019 NLRB policies, she would not have been awarded exception points and may never have received a transplant. The new proposal is a first step in the right direction, but I implore you to continue to examine the data carefully. From my close involvement in the PSC community, I know that PSCers can get very, very sick while holding a MELD in the teens or low 20s. They become frail and weak, and then something acute happens and they can't survive it. A PSCer with a MELD of 30 is an extremely sick individual. Thank you for taking to heart the voices of the PSC community during the last comment session and proposing these changes, but please keep the PSC patient population top of mind. These patients are not on the waiting list due to unhealthy life choices, and at this time there is no other real treatment for their disease. For many, this is their one and only chance - please make sure that they get it. Thank you for all you do; you have to make hard choices that affect everyone on the transplant lists, and you are appreciated! Deanna Vinovich | 03/19/2021 Husband dx 1999. Luckily found PSC Support and have lost so many members who could not get exception points. Changing the policy to easily get more clearly and exception points will be a help to many PSC patients. Thank you for this opportunity to post our support. Linda Waters | 03/19/2021 I have PSC and have been fortunate to be asymptomatic. Through the course of my disease, I have made many friends with PSC. Unfortunately over the last 10 years I have lost many friends who have passed away because their Meld score didn’t reflect how sick they were. All of these friends who have passed away went way too soon. Too many good people have been lost because they were not awarded exception points and they succumbed to this dreadful disease. I am so glad the NLRB is finally making changes for PSC patients. I totally support the proposed changes and truly believe the changes will save lives. Anonymous | 03/19/2021 I fully support added points in selected patients with PSC awaiting transplant because *the MELD score does not adequately reflect the severity of illness before transplant*. I was end stage at MELD 21 and know of at least a dozen other patients who were in the same boat. Three had MELD scores below 20 and were at end stage with severe encephalopathy. One thing we all had in common: our serum creatinine levels were lower relative to the degree of liver damage, thus restraining the MELD score. Dropout rates are higher among patients with PSC. Mike and Eileen Pearlman | 03/19/2021 Thank you for the opportunity to comment. We support the proposed changes to the PSC guidelines. Hopefully, it will be beneficial to future PSC patients awaiting transplant. Our daughter passed in 2013. In the last year of her life (November 2012 to November 1, 2013) she was hospitalized several times. During this period, MELD scores were a frequent occurrence. What struck us was the fluctuation in her scores. In her last year of life, her MELD scores fluctuated from 15 to 39. When the scores were high, it was our impression that Drs. were hesitant to treat her with Vitamin K, as this could impact on the likelihood of her receiving a transplant. Giving her infusions brought her MELD down. This removed her from the top of the transplant list, until her scores went back up following a later retesting. In other words, she had to be kept at the highest risk level as long as possible. She had to border on the edge of dying to keep her MELD score up for the possibility of transplant. On October 17, 2013, our daughter elected to enter hospice, passing on November 1, 2013. Anonymous | 03/19/2021 The proposed changes would help move patients with PSC to a more even playing field in terms of MELD score. This would be helpful as physicians are no longer able to give points at their discretion. Anonymous | 03/19/2021 The MELD for PSCers does not reflect the true illness. I have heard from many PSCers that surgeons are surprised at the damage of PSC livers at transplant because the MELD score did not reflect what they were expecting. Recent statistics show that the dropout rate of PSC transplant candidates with a MELD or PELD score greater than 37 is substantially higher for PSC patients than for other listed liver patients. This data confirms what PSCers have long observed. As a result, PSCers are often encouraged to find living donors because of repeated sepsis, the poor MELD reflection of illness, and/or the added risk of CCA (which is difficult to discern in PSCers). PSCers need the additional exception points from this proposed change. Angie Aldarondo | 03/19/2021 I strongly support the National Liver Review Board's recommendation to change the policy for exception points regarding the frequency of hospital admission and the location of admission. My sister-in-law, who is one of the brightest, kindest, and most wonderful people you'll ever meet, has been living with PSC for many years. Her beautiful boy (my nephew), husband (my brother), and our entire family need her around for a long and full life. She has already suffered, with incredible grace I might add, more than any one person should. Please make the changes necessary so that she can have every chance made available to her. She's the kind of person who makes the world a better place for having been in it, and we need to fight to do all that we can to protect her and all others who are struggling with this terrible disease. Veronica Martinez | 03/19/2021 My sister has rPSC and watching her (and those in the PSC community she has connected with) decline in health, waiting and hoping to be in the very narrow space of being sick enough for the MELD score to align with current thresholds for transplant but not sick enough for it to be too late is heartbreaking. I strongly support these changes and the opportunity for those who desperately need livers to be seen more accurately within the system. Thank you. John Daniels | 03/19/2021 MELD for PSCers fluctuates and does not reflect our true illness. Surgeons are surprised at the damage of livers at transplant because the MELD score did not reflect what they were expecting. PSCers are often encouraged to find living donors because of the added risk of CCA and the poor MELD reflection of illness. PSCers need the additional exception points. Anonymous | 03/19/2021 The MELD for PSCers does not reflect the true illness. I have heard from many PSCers that surgeons are surprised at the damage of PSC livers at transplant because the MELD score did not reflect what they were expecting. Recent statistics show that the dropout rate of PSC transplant candidates with a MELD or PELD score greater than 37 is substantially higher for PSC patients than for other listed liver patients. This data confirms what PSCers have long observed. As a result, PSCers are often encouraged to find living donors because of repeated sepsis, the poor MELD reflection of illness, and/or the added risk of CCA (which is difficult to discern in PSCers). PSCers need the additional exception points from this proposed change. Monika Aldarondo | 03/19/2021 I am a transplant recipient and because of recurrent PSC a probable future candidate for a second transplant. I am in support of the policy change for exception points for PSC patients. Shortly after I was diagnosed with recurrent PSC, I was hospitalized with Bacteremia. In that hospitalization and in subsequent appointments, I have been told repeatedly that I will eventually need a transplant and in the next breath that I probably wouldn’t qualify for another deceased donor via MELD. This change could help, if not me, the sickest of my fellow PSCers. There is always the fear of not making it to transplant. While I was never hospitalized before qualifying for my first transplant, I know others that would benefit from this policy change. Maybe even my future self. Thank you. Thomas Ivacko | 03/19/2021 I am a caregiver for my wife, who was diagnosed with PSC in 2018. As we've learned more and more about PSC, it's become clear to us that the MELD score does not correlate with or accurately represent actual patient health for those with PSC. It's been very distressing to know that transplant decisions are being based in part on such a key metric, when we know the metric isn't giving an accurate picture of a patient. This feels like a situation with an urgent need for remedy, and we're very grateful that you are considering a policy change. Thank you for this opportunity to comment. I strongly support the proposed changes to guidance for patients with PSC. Anonymous | 03/19/2021 I strongly support the proposed PSC guideline changes and am so grateful that the NLRB is listening to the voices of those affected by PSC. PSC is a destructive disease that takes over your body, leaving you sick, intensely itchy, extremely exhausted, and in pain, no matter how healthy you live your life. The level of sickness that PSCers experience is not reflected in the current MELD/PELD score system and it breaks my heart to hear the stories of PSCers who lose their battle with this devastating, cruel disease due to the fact that by the time their MELD score is high enough to be considered for transplant, they are too sick to receive the procedure. Lives could be SAVED by changing the current transplant policy. Imagine that! What a gift it would be if the suggested changes are implemented. John Daniels | 03/19/2021 MELD for PSCers fluctuates and does not reflect our true illness. Surgeons are surprised at the damage of livers at transplant because the MELD score did not reflect what they were expecting. PSCers are often encouraged to find living donors because of the added risk of CCA and the poor MELD reflection of illness. PSCers need the additional exception points. Julie Turk | 03/19/2021 I am a parent of a daughter whom has PSC. She has been on the transplant list since September 2020. Her MELD score does not reflect her need and pray that changes are made so that she doesn't die waiting for a transplant. It is imperative that PSC patients receive additional points to equitably give people with this disease a fair opportunity to receive the gift of life. Please consider this change so that people with this disease are given a fair chance to live. Eric Holman | 03/19/2021 Cholangiocarcinoma, as I understand it, co-occurs often with PSC. And, as I understand it, transplant patient candidates with PSC often have a higher symptomaticity than transplant candidates with other diagnoses at a given MELD score. So I support the NLRB effort to make exception points more accessible to PSC patients with CCA. On a personal note, I would remind NLRB to be aggressive and fair in updating policies to reflect current science. When I was being evaluated for transplant I had experienced 2 esophageal bleeds and recurrent cholangitis, malnutrition and pruritis to the point of suicidal ideation at a MELD of 15. My team advised me that I would do best with a living donor organ, because I would likely not survive the waitlist to get an expanded criteria liver donation. Their explanation was that PSC does not kill the liver in such a way that MELD scores reflect patient experience and mortality. It's just a tad harmful to hear from your physicians that a quorum of rule-makers haven't updated their methods to include a whole category of patients, into which you fall. While not medically relevant, the fact that a PSC patient can take no action to control their likelihood of relapse is a psychological factor which adds to the burden of PSC being underrepresented in MELD. I appreciate this action by NLRD to begin correcting that. I have occasionally joked that I would pay good money to be an alcoholic so that I could have a hand in controlling my outcomes. Please don't leave myself and future PSC patients in a position where jokes like that are relevant. I'll work to create opt-out legislation in my state, you keep up your good work too. Thank you. Mike Maly | 03/19/2021 The data from National Liver Review Board (NLRB) show that the current transplantation guidelines do not account for the complexity and severity of PSC liver disease and that MELD scores often does not accurately reflect the severity of the disease. I support the NLRB’s proposed changes. Our daughter was diagnosed with PSC a few years ago and constantly pray she will progress slowly, but I know she will likely need a liver transplant in her lifetime. I also pray that when she needs a transplant, she will receive one as I know the MELD score does not accurate reflect how severe the disease is or how sick people really are with PSC. Anonymous | 03/19/2021 My son-in-law is on the transplant list for PSC. He works so hard to live a healthy lifestyle and to prioritize wellness, however his disease progression is out of his control. He will likely need a transplant in the future. Because of the current standards, he is much more likely to become too sick or die waiting to be transplanted than any other listed liver patients. We are fearful of losing him to a disease that can be treated by transplant because the MELD scores do not take into consideration the complexities of the PSC disease. I am in support of the policy change.. Judith Ivacko | 03/19/2021 I am a physician who was diagnosed with PSC in 2018. I do everything possible to maintain a healthy lifestyle but am aware that the disease is likely to progress despite my efforts. The data from the National Liver Review Board clearly indicate that the current transplantation guidelines do not account for the complexity of this disease and that the MELD score often does not accurately reflect disease severity in any given patient. I support the NLRB’s proposed changes and encourage continued surveillance of the waitlist mortality and dropout rates as policy changes are made in order to ascertain their effects on these rates. Thank you for the opportunity to provide feedback about this important process. Northwell Health | 03/19/2021 We support the greater inclusion of PSC as an exception pathway. There is no doubt a higher dropout rate for patients with PSC who develop high MELDs. This likely represents the chronicity of their disease and debilitation in relation to other patients with equal MELDs. These patients deserve earlier access. Gwyneth Smith | 03/19/2021 My husband was just recently diagnosed with PSC. He has lived with a host of other autoimmune issues, in which many doctors have been proactive in treatment. However, upon this diagnosis of PSC, we were immediately made aware of the road to transplant may look like should his disease progress. This disease is rare, still in early stages of understanding, and lacking effective treatment. We are finding that it runs its course differently in each patient, and while we may never reach the point where he would be in need of a transplant, the odds suggest otherwise. I would hope that eligibility for transplant would be met with consideration for PSC rather than the standard measures put in place for a blanket of diseases that lead to liver transplantation. We are glad to see consideration and advancements being brought to the table on behalf of all of those living with PSC, and are hopeful that with future research and updates to current policies, that people who are fighting their battle with PSC will have equal opportunity to overcome this disease without the obstacles that are unfairly placed against them. Thank you for your time and consideration! Amanda Pugliese | 03/19/2021 I was diagnosed with PSC in 2013 and my symptoms began to worsen around 2016. At that point the severe itching, jaundice, extreme fatigue, upper right quadrant pain, depression, etc were interfering with my job, relationship, and many other areas of my life. I knew I would not be able to live like this for much longer. My team and I decided it was time for me to get on the list, however, I was told I would likely have to wait a long time due to my low MELD score. I didn’t understand. I was so sick but I was not considered a needy candidate as a result of the unfair guidelines and my diagnosis of PSC. I was lucky enough to find a living donor but many of those with PSC don’t have that opportunity. I’m also scared that my PSC will come back (a very real possibility) and that I will have to suffer again on the list as a second living donor transplant is unlikely. I beg you to reconsider the current guidelines so that people with PSC have their fair chance in receiving a life saving transplant before it’s too late. Nicola Tessier | 03/19/2021 As a post-liver transplant patient that has been greatly affected by Primary Sclerosing Cholangitis, I fully support the National Liver Review Board’s proposed changes to guidance for candidates with PSC. I am a mother with 2 young children at home and hospital admissions are complex and stressful as we try to make the stress and disruption as minimal as possible for our kids, all while advocating for the best care. I have experienced sepsis numerous times and it is an ongoing challenge for me requiring long-term antibiotics. The proposed policy change that PSC patients receive exception points after admission to the hospital 2 or more times within a one-year period with a documented blood stream infection is a necessary change to help those listed for transplant. We need to even the playing field for PSC patients and I’m hopeful this change will be a much needed step in the right direction. While this may not be a cure, it provides a lot of hope for those fighting the good fight daily. Thank you for your consideration. Jonathan Ackerman | 03/19/2021 The current method does not work the way it should. My wife was hospitalized several times a year for PSC and had no quality of life. She was in bed all day every day for the most part. She fortunately received a transplant 2 months ago. The main reason for my support though is that she was the backup person when she received her transplant. The person that was first passed away before they could get the transplant surgery. That’s not OK with me. They were so sick that they did not make it long enough to receive a transplant! They should have received a transplant much sooner and they may have lived longer. Robert Bellanich | 03/19/2021 I am a post transplant PSC patient who suffered with PSC for 18 years with a low meld score. When it progressed 16 years later, I had muscle mass wasting, severe itching, loss of weight and feeling fatigue 24/7. While I was never hospitalized with sepsis, I also never felt that my MELD reflected how sick I truly was which led to my wife putting a petition together out of desperation. To date, over 9000 people have signed in support of giving PSC and PBC patients exception points because we have experienced this first hand.. After my transplant, the picture of my old liver was unbelievable. Today I feel like I have a new engine in me because of the miracle of transplant. Thank god!! This proposal is a first step towards change which I support. Please help us before all our other organs are affected. Edward K | 03/18/2021 As a person living with PSC there is nothing more frightening than the unknown. You make healthy changes to your daily life style in hopes of living a little bit longer but ultimately have no idea if it will matter at all. We live with the ongoing concern of when our health will deteriorate and we can no longer work and care for our families. When will the day come when we have to tell our children that we are sick and they have to watch us suffer and live a life of pain. PSC patients try hard to live each day. We take great care of ourselves and deserve to be treated with equal opportunities to live because we don’t take life for granted. I had a meld score around 12 when I was first diagnosed 10 years ago and it was tough. I was in pain and jaundice. I can’t imagine what it would feel like having a score over 30 and just hoping and praying for a transplant. If PSC patients have more accurate access to transplants that can only be a good thing because we are eager to live. I was a registered organ donor prior to getting PSC and I would have certainly been proud to give my liver to a PSCer knowing they would use it to continue living and appreciating life. Anonymous | 03/18/2021 I am a physician who was diagnosed with PSC about 20 years ago. My mother died of PSC at age 44 and her brother died of PSC about age 59. The MELD disparity in exception points for PSC patients has been a concern for years. I strongly support the proposed change in exception points for PSC transplant candidates from two or more hospitalizations in the past three months to two or more hospitalizations in the past year. Thank you. Marilyn Hall | 03/18/2021 I am elated and saddened with the proposal for exception points for PSC patients!! My husband lived with PSC for 22 years. Major side effects were held at bay with medication. Fatigue was always a factor! From 2016 on, he started to develop more issues. In Oct of 2018 we buried his mom who was 99!! His health really started to decline after that. Jaundice and a forty pound weight gain of fluid that was miserable for him. Large amounts of diuretics helped with this. But then kidney levels had to be monitored. Hand and leg cramps became a thing. But it was the toxin buildup that was the game changer. In January 2019 he underwent pre-transplant testing and was approved for a transplant on April 2nd. We were happy but I could see what a toll all of this was taking. The docs kept telling us that waiting was the worst part. His MELD score was not high enough to bump him on the transplant list. He just kept taking meds to try to level out the hepatic encephalopathy but it was becoming too late. I watched my 6’7” 230 lb husband become so weak, jaundiced, sleeping most of the time, and yet still hoping for another chance at a healthier life! I could go on and on with more stories but the simple truth is that big changes need to be made for PSC patients!! My husband had to be hospitalized for 2 weeks in June. His condition worsened daily. He was LifeLined to another hospital but there was nothing left to do. He died from a perforated bowel and sepsis on June 29, 2019. This was heartbreaking and all I could think was how he might have had a liver transplant if he hadn’t had to wait so long and get so sick. Living with PSC and being a spouse to someone with this disease is so challenging Please please give them a chance at a transplant before they became so very ill. Labs and numbers don’t always indicate how sick a PSC patient is. I can say this from personally witnessing my beloved husband and his courage to try and live. My husband was 67 at the time of his death. Angrla Solemsaas | 03/18/2021 Please consider these changes for all of the PSC patients and their families. I still have a young family to raise. Thank you for your consideration. Mindy deMedeiros | 03/18/2021 My nephew was diagnosed with PSC at a young age. Not only has it been challenging for him and his family, navigating treatment options has been an exhaustive and lonely process for my sister. My nephew now in his early 20s has constant awareness of the state of his liver and it would be a relief for him and his family to know set a donor transplant is possible. My nephew has done nothing toCause this disease. Quite the contrary he has spent most of his life reading up on the disease and making safe choices that will prolong the life of his liver. He and other PSC patients deserve to be high up on the transplant list. Dana Olson | 03/18/2021 I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My brother in law is on the transplant list for PSC. He works so hard to live a healthy lifestyle and to prioritize wellness, however his disease progression is out of his control. He will likely need a transplant in the future. Because of the current standards, he is much more likely to become too sick or die waiting to be transplanted than any other listed liver patients. We are fearful of losing him to a disease that can be treated by transplant because the MELD scores do not take into consideration the complexities of the PSC disease. Please support my brother in law and the community of people affected by PSC. Anonymous | 03/18/2021 Statistics show that patients with PSC and advanced stage liver failure are not sufficiently accounted for in the current guidelines. The updated changes are one crucial step, among many others needed, to begin to properly account for the complexities of this disease in relation to organ availability. In addition, automatic approvals for CCA exemptions are also important in saving those who are battling this difficult and under researched form of cancer. Anonymous | 03/18/2021 Every day we live with the joys brought by our grandchildren. But, every day we also deal with the difficulties faced by one grandchild who was diagnosed early in life with PSC. We’ve learned a great deal about the disease and the unrelenting course it takes. A liver transplant is a real possibility in our future, and knowing that the current MELD score system produces significantly higher waitlist mortality and dropout rates for PSC patients - and the progressive debility in living that can affect them while waiting - is just unfathomable. We strongly urge the NLRB to allow exception points to be added to the MELD score for PSC patients. Bobby Olson | 03/18/2021 I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My brother is on the transplant list for PSC. He works hard to live a healthy lifestyle and to prioritize wellness, however his disease progression is out of his control. He will likely need a transplant in the future. Because of the current standards, he is much more likely to become too sick or die waiting to be transplanted than any other listed liver patients. We are fearful of losing him to a disease that can be treated by transplant because the MELD scores do not take into consideration the complexities of the PSC disease. Please support my brother and the community of people affected by PSC. PSC Partners Seeking a Cure | 03/18/2021 I was glad to read of the proposed changes to the MELD guidelines that recognize PSC, and I greatly support the proposed changes. My mother lived with PSC for many years but for too many, we did not know what inflicted her. I know she is cheering in Heaven to see that PSC is getting to be more recognized and understood! — Nicole De Santis Anonymous | 03/18/2021 As a patient who had sepsis twice and a massive hemorrhage that I almost died from, exception points were critical in my ability to get a transplant. Unfortunately, I wasn't given them until after I suffered serious trauma that could have been prevented had the sepsis been enough to earn the exception points. Even then, I needed to accept a diseased (hep c+) organ because with my 5 exception points, my MELD was still only 22. I am lucky and blessed to be alive. Gary Olson | 03/18/2021 "I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My son is on the transplant list for PSC. He works so hard to live a healthy lifestyle and to prioritize wellness. However his disease progression is out of his control. He will likely need a transplant in the future. Because of the current standards, he is much more likely to become too sick or die waiting to receive a transplant than any other listed liver patients. We are fearful of losing him to a disease that can be treated by transplant because the MELD scores do not take into consideration the complexities of the PSC disease. Thank you for taking this into consideration and adjusting the rules on the scores in fairness to PSC patients. Shay Olson | 03/18/2021 I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My son is on the transplant list for PSC. He has always lived a healthy lifestyle and especially so after his diagnosis 8 years ago. But like everyone with PSC, no matter how hard he works to prioritize his wellness, the progression of the disease is beyond his control. He will likely need a transplant in the future. Unfortunately, because the current calculation of MELD scores do not take into consideration the complexities of the PSC disease, he is much more likely to become too sick or die waiting to receive a liver transplant than any other listed liver patients. We are fearful of losing him to a disease that can be treated by transplant. Please support my son and all those affected by PSC. Bobby Olson | 03/18/2021 I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My brother is on the transplant list for PSC. He works hard to live a healthy lifestyle and to prioritize wellness, however his disease progression is out of his control. He will likely need a transplant in the future. Because of the current standards, he is much more likely to become too sick or die waiting to be transplanted than any other listed liver patients. We are fearful of losing him to a disease that can be treated by transplant because the MELD scores do not take into consideration the complexities of the PSC disease. Please support my brother and the community of people affected by PSC. Dana Olson | 03/18/2021 I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My brother in law is on the transplant list for PSC. He works so hard to live a healthy lifestyle and to prioritize wellness, however his disease progression is out of his control. He will likely need a transplant in the future. Because of the current standards, he is much more likely to become too sick or die waiting to be transplanted than any other listed liver patients. We are fearful of losing him to a disease that can be treated by transplant because the MELD scores do not take into consideration the complexities of the PSC disease. Please support my brother in law and the community of people affected by PSC. Anonymous | 03/18/2021 I am writing in strong support of the proposal to shift how PSC patients are considered for transplant. The current MELD scoring system is incredibly unfair to patients, whose scores often drop with interventions pre-transplant despite the progressive disease. Our son was diagnosed with PSC at age 4 and we are incredibly fearful about walking the line to transplant without him becoming too sick to actually receive one. There are too many PSC patients who fall into that gap and lose their chance for a transplant despite a progressive primary disease that should be evaluated differently than secondary liver disease. Please consider making this change to allow for a more fair system for PSC patients. Kelli Klippenstein | 03/18/2021 I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. I have a family member on the transplant list for PSC. He works so hard to live a healthy lifestyle and to prioritize wellness, however his disease progression is out of his control. He will likely need a transplant in the future. Because of the current standards, he is much more likely to become too sick or die waiting to be transplanted than any other listed liver patients. We are fearful of losing him to a disease that can be treated by transplant because the MELD scores do not take into consideration the complexities of the PSC disease. Please support my family and the community of people affected by PSC Kendra Pearson | 03/18/2021 I was transplanted in 2014 because of PSC. My husband had to donate his liver to me because I was too low on the list to receive a liver. I was told I had three months to live so he donated. Now in 2021 I have recently been relisted due to rPSC. I again am too low on the list and I am very very sick. I have been unable to find a live donor this time. I have to be on strong antibiotics everyday because if I do not I get Cholangitis. Nothing can be done for me. My bile ducts have too much narrowing and no blockages. The side effects from all the antibiotics are starting to bring on a whole other set of problems not to mention the risk of becoming resistant to them. If I'm not on antibiotics I have to be hospitalized (already have twice and once I became septic). How can I go on like this. I either live in a hospital or take antibiotics until I end up in hospital. Great options. I'm only 41 and have a 15 and 12 year old who need me. I can guarantee that if someone were to hang out with me for a day or a week, they would see I'm as sick as patients that are being transplanted but my meld numbers just don't reflect it. Please help patients like me who are in desperate need of livers and see us as not just a few numbers that don't truly reflect how sick we really are. I'm not going to live much longer if something isn't done for our disease! Nathan Olson | 03/18/2021 I have been listed for transplant since I was first diagnosed with PSC in 2013. I maintain a healthy lifestyle, track my health closely, eat well, exercise, and don't drink alcohol. Nonetheless, I have little to no control over my progression worsening. I will likely end up becoming sick enough to need a transplant in the future, and under current MELD calculation rules, I may not be eligible until I am very sick. Liver transplants are the only proven treatment for PSC but are less effective the sicker the patient becomes. The current MELD rules do not apply fairly to the treatment of PSC, and for the sake of myself, my partner, and my kids, I am strongly in support of updating the rules to better reflect the realities of PSC. Sara Olson | 03/18/2021 I support that PSC transplant candidates receive exception points if they are admitted to the hospital two or more times within the last year. My partner is on the transplant list for PSC. He works so hard to live a healthy lifestyle and to priorize wellness, however his disease progression is out of his control. He will likely need a transplant in the future. Because of the current standards, he is much more likley to become too sick or die waiting to be transplanted than any other listed liver patients. We are frequently fearful of losing him to a disease that can be treated by transplant becasue the MELD scores do not take into consideration the complexities of the PSC disease. Please support my partner and the community of people affected by PSC. Anonymous | 03/18/2021 As a PSC patient I strongly agree with changing the MELD score guidelines for transplant. PSC patients need a voice and accurate calculation of MELD scores Kelleigh Youngclaus | 03/18/2021 I am in strong support for updating the liver allocation requirements to include exception points to the MELD score for patients with Primary Sclerosing Cholangitis. I have been a witness to the disease both as a researcher and family member. Working with patients as a research coordinator at The Resnek Family Center for Primary Sclerosing Cholangitis (PSC) Research at Brigham and Women's Hospital (BWH), in addition to having an aunt diagnosed with PSC late in life, it is clear that MELD scores are not always indicative of how in need PSC patients are. This policy change is essential to improving patient dropout rates. Region 11 | 03/18/2021 Region 11 sentiment: 0 strongly support, 11 support, 5 neutral/abstain, 0 opposed, 0 strongly opposed During the meeting there were comments voiced during the discussion, submitted online and submitted as comments from the OPTN representatives. Overall, there was general support for this proposal. One attendee thanked the committee for the work and commented that they appreciated the policy clarifications. Another attendee recommended more precise guidance for appropriate PELD scores or to provide a more reliable system to assess waitlist mortality in children. The attendee went on to recommend more standardization for listing pediatric candidates. Another attendee added that with the pediatric exceptions being higher than 50% of those listed, the PELD score does not work for these patients. One attendee commented that ischemic cholangiopathy should be included in the secondary sclerosing cholangitis group as a safety net for DCD recipients. Another attendee was concerned about using the number of days a candidate is hospitalized since this can lead to gaming the system. Anonymous | 03/18/2021 I was diagnosed with PSC in 1991 when I was thirteen. In 2015, during the last trimester of my 2nd pregnancy, I went into rapid liver failure due to PSC. For about 3 months, our family experienced the torturous uncertainty of 1: whether our baby would be delivered safely and 2: whether I'd survive to care for our kids. Good news -- an organ donor saved my life 6 weeks after my son was safely born! Six years later, I have been able to parent my 8- and 6-year old children through first steps, first days of school, and now even a first pandemic. I am alive today because of organ donation and the incredibly fast and responsive measures taken by my medical transplant team and my region's organ procurement organization. In cases like mine - days can make the difference between life and death. I heartily support these changes to the NLRB guidance document that will streamline approvals and ease access to organ donation for future PSC patients. Deborah Wente | 03/18/2021 My daughter was dx with PSC when she was 10 years old. She is now 25 and suffers daily from the effects of this disease. She is not yet eligible for a transplant, but it has been one of the great fears of my life that she will be so sick before she is eligible that she will not survive. She's only 25 and is getting married this year. My heart breaks for her. Darin Mayne | 03/18/2021 I am just going to be forthright, primary schlerosing cholangitis needs exception points added to the meld score. It is through no fault of their own they got this wretched disease and should not have to wait behind someone who made bad life choices knowing full well the consequences of that action. I know someone that keeps hearing they look good for a 20 meld score, their liver and bile ducts disagree and will statistically turn cancerous or kill them . It is insane that with all the knowledge of liver disease types and causes that we are not helping the few who never asked for liver disease. What a shame . Thomas Walsh | 03/18/2021 I would like to state my support for a change that would allow PSC transplant candidates to receive exception points if they are admitted to the hospital two or more times within the last year. My son was diagnosed with PSC at age 7 and is now 9 years old. While he is doing well at this time we know that with his disease progression he will eventually need a transplant. Please consider this proposal as it could mean life or death for my child. Too many PSCers die before they receive a transplant due to the current scoring system. Thank you! Anonymous | 03/17/2021 I have PSC and I tell friends that I have a long-term lease on my liver. When that lease is up, I hope that my MELD score will take into consideration those factors that are specific to PSC'ers. Exception points for PSC patients admitted to the hospital two or more times within the last year would be a good start. Anonymous | 03/17/2021 I am in strong support for updating the liver allocation requirements to making an exception to the MELD score for patients with Primary Sclerosing Cholangitis . My son was diagnosed at age 20, and making this change would better serve his health needs. Remember, this disease has no cure. Anonymous | 03/17/2021 I'm 34 years old and was diagnosed with PSC 8 years ago. I've also been living with ulcerative colitis and now Crohn's for 21 years. I've been living with active diseases for so much of that time, trying to do everything I could do to feel better (change my diet, modify stress, try lots of different medicines, get more sleep, etc); all to end up with hospital stays, secondary infections from medicines, and a few surgeries along the way. The situation is really out of our control. My conditions are more stable now than they've been in years, but I'm also pregnant and hope to be around for many many years to celebrate and spend good quality time with our growing family. Anonymous | 03/17/2021 Thank you for the opportunity to comment on the proposed changes for the transplant criteria. My son is a PSCer diagnosed at age 20. He is now 35 with two young children and dealing with progressive disease and abnormalities on both FISH and cytologies. I/we wholly support the proposed changes in both Meld and cholangiocarcinoma criteria for transplant. Thank you so much for addressing this unmet need! Anonymous | 03/17/2021 Hello, I am writing to strongly support these updates. As a grateful liver transplant recipient due to a longstanding bile duct disorder, PSC, I am keenly aware that the MELD score does NOT represent the severity of the liver disease progression in PSC. For this reason, folks with PSC can be significantly sicker than in other disorders more adequately measured by the MELD score by the time transplantation criteria is met using the MELD score. In addition to dropouts due to severe disease, folks that are sicker when transplanted have higher complications and longer recovery times. Use of a metric that does not adequately reflect the degree of liver disease for transplantation candidacy seems spurious at best, and significantly disadvantages folks with PSC. This seems grossly unfair in the allocation of the precious limited supply of organs for the PSC patient community. Other measures should be added, as the proposal does, to augment the metric of illness for consideration for transplantation. This will help level the field for PSC patients opportunity for transplantation. PSC transplant recipients have one of the highest success rates post-transplant, and this disorder should not be at a disadvantage per the current system approach and use of the inadequate MELD score, which does not capture degree of liver damage in PSC. (I also strongly support the removal of age limits, as there is significant variation in folks' health no matter their age; inclusion of an age limit seems arbitrary for this reason.) Thank-you for your time and consideration. PSC Partners Seeking a Cure | 03/17/2021 PSC Partners Seeking a Cure supports the National Liver Review Board (NLRB) proposed changes to guidance for candidates with PSC/SCC (sclerosing secondary cholangitis). PSC Partners, a 501(c)3 non-profit organization, provides education and support to PSC patients, families, and caregivers and raises funds to research causes, treatments, and cures for primary sclerosing cholangitis (PSC). We hear stories from our community of how the MELD score often does not reflect how seriously ill PSC patients are, and how the MELD score can drop after an ERCP or radiological intervention, yet the individual continues to live with progressive debility, and unrelenting, recurrent symptoms. We appreciate the NLRBs review of the waitlist dropout rates which showed that the PSC waitlist dropout rate was significantly higher for PSC candidates with a MELD or PELD score of more than 37 than for non-PSC listed transplant candidates. The NLRB proposed policy change that PSC patients receive exception points after admission to the hospital two or more times within a one-year period, and that candidates must be admitted to the hospital with a documented blood stream infection, or evidence of sepsis including hemodynamic instability requiring vasopressors, is an important step to help address the increased proportion of PSC patients who dropout due to death or being too sick to transplant. PSC Partners agrees that this policy change is essential. We would also like to ask the NLRB to continue to annually monitor the waitlist mortality and dropout rates, including the waitlist mortality rates for those with high MELD scores. We hope to be able to see data in a year’s time to measure if and how this policy change has affected waitlist dropout. Again, we thank the NLRB for this positive change for PSC and SSC patients. We look forward to on-going efforts to help all liver patients receive transplantation. Matt Evans | 03/17/2021 I fully support the proposed changes to exception points for PSC and CCA patients. My wife was diagnosed with PBC in 2016 at age 42. By the time of diagnosis, he had cirrhosis and other complications that required him to go straight to transplant evaluations. My wife is fortunate enough to not need a liver right now, but she will likely need one in the future. The PSC/PBC community have long heard the argument against providing exception points for PSC/PBC patients due to them having a lower waitlist mortality, but recent analysis suggests that the waitlist mortality is actually much higher. The proposed changes to the exception points for PSC/PBC would help these patients obtain the scores needed to get a full liver from a deceased donor. Additionally, their transplants need to happen in a timely manner and within the parameters of their treatment protocols. Giving PSC/PBC patients a clearer path to exception points could improve their odds at getting a transplant. I am so thankful the NLRB is considering these important changes. These patients need a shot at living full and healthy lives. Better access to deceased donor transplant through exception points would be a huge step in the right direction. James Harvey | 03/17/2021 As a young person with PSC facing a potential liver transplant in the future the reduction in requirements for ICU admission vs hospital admission is massive. I was diagnosed with PSC 2018, the same year my wife and I were married. We are currently expecting our first child and anything that increase the potential of me being around longer and see our baby boy grow up is huge. The idea of having to be so close to death before moving up on the transplant list is scary. I know the process is as fair as can be but from the data shown I think it speaks for itself in the risk associated with patients with PSC based MELD scores >37. KEVIN O'DONNELL | 03/17/2021 I am coming up on my 3rd anniversary of being status 1B/4 on the heart transplant waiting list. I feel the move to the new allocation system is missing an important consideration. I am in a good donor area, and a lot of donor hearts go to other areas far away now, to areas that have a very poor organ donor registration percentage (eg. New York as an example). While I understand there are candidates that will always be sicker than me, there seems to be no consideration of time waiting. I feel a a candidate should be moved up a status level after a certain amount of time on the waiting list. Every day that goes by, I feel the Lvad I have could have a problem/complication that could seriously impact my heath and transplant candidacy. Region 7 | 03/12/2021 Region 7 sentiment: 2 Strongly Support; 4 Support; 4 Neutral/Abstain; 0 Oppose; 1 Strongly Oppose. During the meeting there were comments voiced during the discussion, submitted online and submitted as comments from the OPTN representatives. Overall, there was general support for this proposal. One attendee mentioned the pediatric growth failure assessment, and if there is potential to factor in the presence or absence of moderate or severe ascites. Another attendee asked if the hospitalization for PSC require bacteremia. Two other attendees stated that, it seemed like documented bacteremia is needed. There were several additional comments made. One attendee mentioned that growth failure has a clear definition, but nutritional insufficiency isn’t as clear and suggested more guidance on what to put into these guidelines. Another attendee commented that, it did not seem reasonable for children to have exceptions leveled out to MMAT competing with adults for HCC. They added, it would give the pediatric patients a great disservice. They also stated that a large number of transplants are split transplants, and then the right lobe will go to an adult. They also added that this would take away organs from children and decreases number of split organs. Another attendee also commented on the 5 days’ hospitalization requirement for the portal hypertension criteria. Stating that in the past, they have tried to avoid hospitalization duration requirements because that could lead to longer hospitalizations than might be required, simply to meet requirements. Adding that hospitalization duration is impacted by many things outside of acuity of illness. OPTN Pediatric Transplantation Committee | 03/11/2021 The Pediatric Committee thanks the OPTN Liver Committee for the opportunity to provide feedback on their Public Comment proposal. The Committee provided the following feedback: The Committee agrees with the proposed changes and cited this project as an example of productive collaboration between the Pediatric and Liver Committees. Christine McKinney | 03/11/2021 Please allow exception points to MELD scores for patients with PSC. The severity of illness is not reflected in their scores. They are at higher risk of death while waiting for transplant. They are at higher risk of developing cancer and not being eligible for transplant. Please review the criteria for these patients. Region 6 | 03/09/2021 Region 6 sentiment: 7 strongly support, 17 support, 5 neutral/abstain, 0 oppose, 0 strongly oppose. The region supports this proposal. One attendee recommended decreasing the hospital admission for PSC proposal from 12 months to 6 months. A member asked if patients with metabolic disorders could be given a pathway for additional priority if they are a domino donor, since they are not taking a liver from the organ pool. A couple members shared their appreciation for these updates and that the committee reached out to different stakeholders in formulating them. An attendee commented that while PSC patients may have been disadvantaged by the previous iteration of the NLRB, these new recommendations might be a bit too loose. Region 8 | 03/09/2021 Region 8 sentiment: Strongly support-2, Support–12, Neutral/abstain-2, Oppose-0, Strongly oppose-0. No comments or questions. Anonymous | 03/03/2021 My 35 year old son was diagnosed at 21 with PSC. He’s now married and a father of two young children. As the progression is different with everyone, he’s had ups and downs until the past few months and now his PSC is progressing rapidly despite treatments and meds. He will more than likely be listed soon and I am asking that exception points for PSC be highly considered. These are strong people with an autoimmune disease who have done nothing to destroy their livers. Please make this change! Debbie Nemeth | 03/03/2021 As a live liver donor and a spouse to a liver graft recipient, who was told he would die from his symptoms (symptoms similar to Primary Sclerosing Cholangitis, which we were told could only be cured through a liver transplant) before his MELD score reached the height required to receive a cadaver liver, since the MELD score calculation made no exception for his symptoms, I am in strong support for updating the liver allocation requirements to making an exception to the MELD score for patients with Primary Sclerosing Cholangitis and similar diagnosis. To be told your extremely debilitating, life altering and likely deadly symptoms can only be cured/improved by a liver transplant and that your spot in line for that liver transplant depends solely on a MELD score that does not take your specific symptoms/diagnosis into consideration is the equivalent of being told, "There is a solution for you, but because the system doesn't recognize you, there's no hope for you and you'll likely die." This should not be. As we grow in knowledge, we should be improving our system and our process. Given what we know now, the liver allocation process should start considering and providing exceptions for diagnosis that can only be cured/drastically improved by a liver transplant outside of what the current MELD score process accounts for. No longer is it simply one's bilirubin, creatinine, INR and sodium levels that determine an urgent need for a liver transplant. There are other factors and other symptoms that are just as deadly and equally can only be resolved through a liver transplant and we MUST start considering and making exceptions for those, too. This is personal for me and for so many individuals and their loved ones in this position. When we know better, we should do better. And we need to start considering and providing exceptions for things outside the basic "bilirubin, creatinine, INR and sodium" counts. Julie Duncan | 02/28/2021 I fully support the proposed changes to exception points for PSC and CCA patients. My husband was diagnosed with PSC in 2015 at age 30. By the time of diagnosis, he had cirrhosis and other complications that required him to go straight to transplant evaluations. Like most patients with PSC, he was advised to find a living donor due to having a relatively low MELD and the risks associated with waiting for a high enough score to get a deceased donor transplant. My husband was lucky to find a living donor, but unfortunately needed a second transplant less than eighteen months later due to cholestatic graft failure. The PSC community has long heard the argument against providing exception points for PSC patients due to them having a lower waitlist mortality, but I have always wondered what those numbers would be if PSC patients weren't driven down the path of LDLT. Would their waitlist mortality be higher? I think the staggering numbers provided in your literature about waitlist dropout suggests that there could be something to this theory. The proposed changes to the exception points for PSC would help these patients obtain the scores needed to get a full liver from a deceased donor-- and a full liver with a full biliary tree logically seems like a better choice for patients with a bile duct disease that carries a significant recurrence rate. I also support the expansion of the criteria for the automatic allocation of exception points for patients with hilar CCA. These patients already face limited access to transplant due to the relatively small number of centers that are willing to treat them. Additionally, their transplants need to happen in a timely manner and within the parameters of their treatment protocols. Giving them a clearer path to exception points could improve their odds at getting a transplant and give them a better shot at beating their horrid cancer. I am so thankful the NLRB is considering these important changes. My husband has been doing amazingly well for the past three and a half years, but on his last ERCP and MRCP, there were findings suggestive that his PSC is returning. I hate this disease. No one asks for it to come into their home or for the destruction it causes. These patients (often very young) need a shot at living full and healthy lives. Better access to deceased donor transplant through exception points would be a huge step in the right direction. Stephen Enderton | 02/27/2021 I was questioned about my comment by a supporter so let me clarify. Advocating for research that will better identify the people that are in the greatest need of a transplant is one thing but to advocate for a special exemption for everybody with a certain disease is simply splitting the liver transplant community. I agree that the MELD score leaves some people out but giving special consideration to a subgroup of people simply because they have PSC does not address the real issue. It simply provides a subjective advantage to some while excluding others. What is needed is better objective testing to determine need for a liver that addresses need across all subgroups Anonymous | 02/27/2021 My 35 year old son was diagnosed at 21 with PSC. He’s now married and a father of two young children. As the progression is different with everyone, he’s had ups and downs until the past few months and now his PSC is progressing rapidly despite treatments and meds. He will more than likely be listed soon and I am asking that exception points for PSC be highly considered. These are strong people with an autoimmune disease who have done nothing to destroy their livers. Please make this change! Lezlee Peterzell-Bellanich | 02/27/2021 Thank you to UNOS for really taking a look at how some changes in the liver allocation affect certain groups of people and trying to "even the playing field." If we had more donors, we would not be scrambling to distribute precious organs. While many people, including myself, feel that the MELD score does not always indicate how sick someone is from their liver disease, this is the accepted barometer. People living with PSC and (please include PBC) often suffer years, sometimes decades, with fatigue, itching, jaundice, etc. before they qualify for transplant. The current policy for doctors to obtain exception points for their patients is way too restrictive. This proposed policy is an important step in the right direction. Instead of having to be admitted to the ICU twice in three months with sepsis to obtain exception points, if patients are admitted to the hospital twice in one year they would qualify. This is more realistically obtainable so I support this. Eileen ODonnell | 02/27/2021 I am writing to support the NLRB revising it's MELD score recommendations to provide exceptions for liver transplants in pt's with PSC. PSC is different from other liver diseases. Often pt's with PSC do not have a MELD score that reflects the severity of their illness and therefore excludes them from transplant consideration. PSC therefore requires these MELD score exceptions. Thank you. Stephen Enderton | 02/26/2021 How will this affect other people on the list? Does it just change who will die in a subjective way? Exceptions are important but I don't know enough to know if PCS needs an exception. Isn't this really the kind of issue that should be discussed and decided by doctors and not by a petition? Elizabeth Hathaway | 02/26/2021 A patient should not have to be on death's door to be a candidate for liver transplant. This needs to be reviewed and modified. Region 5 | 02/19/2021 Region 5 sentiment: 7 strongly support, 18 support, 10 neutral/abstain, 0 oppose, 0 strongly oppose. During the meeting there were comments voiced during the discussion, submitted online and submitted as comments from the OPTN representatives. Overall, there was general agreement and support for this proposal. One member stated they were confused about the 3-cm criteria for cholangiocarcinoma, stating it used to be an exclusion, and now it is a criterion for inclusion. There is a need for clear language for what constitutes a hilar mass. It is important to be clear for listing and exception. The member asked how would it be defined and are there imagining requirements? The member commented that the language should be clearer and that is one thing that has kept so many exception requests at bay is a need for tissue. The member stated that criteria was fairly strict and kept requests low. Region 3 | 02/18/2021 Region 3 sentiment: Strongly Support-2, Support-14, Neutral/abstain- 6, Oppose -0, Strongly oppose- 0. During the meeting there were comments voiced during the discussion, submitted online and submitted as comments from the OPTN representatives. A member commented that the use of a grandfather clause has been inconsistent across changes to status exception policies. Region 4 | 02/04/2021 Region 4 sentiment: 5 strongly support; 12 support; 2 neutral/abstain; 0 oppose; 0 strongly oppose. During the meeting there were comments voiced during the discussion, submitted online and submitted as comments from the OPTN representatives. Overall, there was general agreement and support for this proposal. One attendee suggested changing requirement for PSC/SSC to 2 hospitalizations within 6 months instead of 1 year. Another attendee suggested providing better guidance on exceptions for ischemic cholangiopathy after DCD transplantation. Amber Breuer | 02/01/2021 My husband was diagnosed with PSC when he was in his early twenties. He was fortunate to have a living donor transplant on June 20, 2016. At that time, he was not on the waitlist and we were told that he would likely die before his MELD would be high enough to get a donor liver. Thankfully his mom was accepted as a living donor, and we have cherished the milestones we've experienced since then (marriage and the birth of our first child). Unfortunately, PSC has returned in his new liver, and now we find ourselves at the beginning of his second transplant journey. PSC does a terrible number on the body, and MELD scores rarely indicate how sick the patient actually is. Unlike many liver diseases, the MELD score can fluctuate from week to week. In the last two months, my husband's MELD has been as high as 30 and as low as 14, despite the fact that he is deteriorating rapidly. Because of this, I believe that PSC patients should qualify for exception points. Patients who have PSC did nothing to bring on the disease, and spend their lives fighting this horrible monster. They deserve exception points that will help their MELD score accurately reflect how sick they truly are. Renee Bacote | 01/31/2021 I would ask that Primary Billiary Cirrhosis (PBC) be included as well. THANK YOU!!! This will help thousands of people living with PSC who are in need of a transplant.