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Guidance Addressing the Use of Pediatric Heart Exceptions

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What is current practice and why address it?

In June 2020, the Organ Procurement and Transplantation Network (OPTN) Board of Directors approved a policy creating a National Heart Review Board (NHRB) for pediatric candidates. It was created to make sure pediatric heart exception requests are reviewed by those with pediatric heart expertise and to better inform decisions. The Heart Committee created a guidance document to provide more information on what reviewers should consider when making decisions on exception requests.

Guidance Addressing the Use of Pediatric Heart Exceptions

Dr. Shelley Hall, Chair of the OPTN Heart Transplantation Committee, reviews the Guidance Addressing the Use of Pediatric Heart Exceptions.

Terms you need to know

  • Exception Request: When a candidate does not meet the requirements for a particular status, but their transplant program believes they are at a similar risk of death while waiting for transplant and have a similar potential for benefit after receiving a transplant, they may ask that the candidate be placed at that higher status. These requests are reviewed retrospectively by the review board.
  • National Heart Review Board for Pediatrics: Board in charge of reviewing exception requests for pediatric heart candidates. Nine pediatric heart specialists from across the country review and make decisions on whether to approve or deny a request.

Click here to search the OPTN glossary

What’s the guidance?

  • Provides information to better inform NHRB members’ decisions on pediatric heart exception requests
  • Guidance is provided on the following pediatric heart diagnoses
    • Dilated cardiomyopathy
    • Hypertrophic or restrictive cardiomyopathy
    • Single ventricle heart disease
    • Coronary allograft vasculopathy and retransplant

What’s the anticipated impact of this document?

  • What it’s expected to do
    • Create more consistency in NHRB exception request decisions
    • Help transplant programs better determine whether to submit an exception request
    • Help transplant programs provide more important information in their exception requests
    • Maintain flexibility for NHRB reviews of unique cases
  • What it won’t do
    • Will not change policy for exception requests

Themes to consider

  • Usefulness of guidelines
  • Ways to notify pediatric heart transplant community about guidelines


Status: Board Approved

Sponsoring Committee: Heart Transplantation Committee

Strategic Goal: Provide equity in access to transplants


Sara Rose Wells


Briefing Paper

Policy Notice


David Rosenthal | 08/16/2020

Thank you for the opportunity to provide feedback on this document. It is a timely and important project that I support strongly. However, there are specific areas in which I believe this work could be improved, both for clarity and content. 1. Explanatory notes p 6 last paragraph suggest that purpose of the guidance is for under 5kg and 10 kg is to prevent situations in which a candidate is given a VAD just to achieve a higher status for transplant. There is no evidence that this practice exists, or is a significant problem. The guidance is developed because some centers are not comfortable placing VADs in smaller patients and a balance is being struck between access to care for patients at such centers, and the overall donor supply. 2. Category 1. The formatting is ambiguous with respect to the sub criteria. It is stated that a candidate must meet all of the following criteria, but is not clear what is a criteria and what is a sub-criteria. The language and formatting should not be ambiguous on such a crucial matter. 3. For DCM less than 5 kg, line 42, page 13. Continuous infusion of milrinone at 0.5 mcg/kg-min is not a high dose inotropic infusion in current practice. It is virtually a required therapy for DCM in the pediatric ICU. For 1A, a higher bar should be set. The category of single high dose infusion should be eliminated as it does not reliably identify a group of patients equivalent to other 1A risk categories. 4. For DCM less than 10 kg, line 42, page 13, the same applies, but in addition, the results from VAD support in this group are sufficiently reliable that I would question the need for this criteria to remain in the document. 5. For older patients, line 44-51, page 13. The presence of arrhythmias is not generally regarded as a contra-indication to VAD support, and in fact, this condition most commonly improves with placement of MCS and should therefore not be considered a reason for 1A exception. 6. For Category 2, line 62, page 14, it would be useful to specify an approximate time frame for the realtionship between the episode of sudden death or the arrhythmia or syncopal events, in relation to the exception request. 7. For Category 3, line 69, page 14, would this criteria apply to all Fontan patients, irrespective of the patient age (so long as the patient was pediatric). In other words, would a 7 yo Fontan qualify, or is this meant to be limited to adolescent patients, in whom the comparison to adult listing status is more incongruous. With the exception of clarifying this point, I strongly support this exception listing status for this population, who generally do not respond as effectively to inotropic support as do DCM patients, and who also have inferior results with MCS as compared to DCM patients. 8. For Category 4, line 87, page 15. This population is urgently in need of higher listing status and I support the effort to define patients at particular risk. However, the criteria as listed are somewhat problematic. First, is it required that the patient be admitted to the transplant hospital? It does not appear in the exception guidance and should be clarified. Second, given the nature of coronary allograft vasculopathy, I doubt that anyone could reliably diagnose triple vessel disease, and further doubt its relevance to prognosis. Third, given that patients with this diagnosis may undergo catheterization after a period of medical optimization, including vigorous diuresis and perhaps inotropic support, I do not believe that the absence of “restrictive hemodynamics” on catheterization represents a population that is distinct from those patients identified as such. The criteria as written incentivize centers to measure hemodynamics under suboptimal conditions in order to qualify for an exception. Furthermore, such patients frequently have multiple catheterizations; is it legitimate to select the one that supports restrictive hemodynamics? As an example, a patient may present with fluid overload and have a catheterization that demonstrates restrictive physiology. Then aggressive diuretics are initiated and fluid limitations imposed. A repeat catheterization is performed to assess efficacy of this approach and shows normalized hemodynamics. What is the center intended to report? This could be improved by expanding the criteria to include diastolic heart failure with frequent admissions for therapy, without diluting the severity of the criteria. 9. In response to the feedback questions, I believe the criteria are nicely inclusive of the relevant contra-indications for VAD support, perhaps even a bit overly inclusive. I do not think they need to be further broadened. As concerns sensitization and CAV, there is insufficient consensus as to how to measure sensitization for this to be included in the 1A exception criteria, in my opinion.

Seth Hollander | 08/21/2020

I would like to echo all of the comments offered by Dr. Rosenthal. As a rotating member of exception request review committee, I strongly support guidance for pediatric programs seeking exceptions. However, the guidance much match best practices and not further promote inequity in the system. In particular, I would like to emphasize my disagreement with two points: 1) Supporting exceptions in children <10 kg who are not on VAD support. Our center routinely places VADs in children 5-10 kg, as do other high-risk implanting centers. Such an exception would disadvantage these patients and disincentivise centers from contenting this practice. Instead, centers who have a patient who would otherwise received advanced therapies at a more experienced center, should seek out transfer of the patient to that center so as not to disantangate them from both status 1A (on VAD) as well as the best treatment options available to them. 2) Milrinone of 0.5. As Dr. Rosenthal mentioned, milrinone of 0.5 is not "high dose" and is used standardly, even in "stable" patients who are feeding intolerant at our center. Moreover, the institution and dose of inotropes is often determined by the ICU not necessarily the transplant physician, who may disagree with that choice. Hence, milrinone 0.5 is too broad a brush to be used as a determinant of illness severity and/or instability equivalent to the need for VAD support. 3) Arrhythmia is rarely, if ever a contraindication to VAD support. In fact, it is often an indication to implant a VAD as arrhythmia often improves after implantation.

Region 4 | 08/26/2020

Region 4 vote: 5 Strongly Support, 10 Support, 8 Neutral/Abstain, 0 Oppose, 1 Strongly Oppose Region 4 supported the proposal. During the discussion, one attendee commented that there should be more details on what would be considered a VAD contraindication for patients in the 5-10 kg range and one attendee suggested the guidance be revised based on broader pediatric heart transplant consensus.

Anonymous | 08/26/2020

This proposal serves to address two important deficiencies in the current system. 1. It is nearly impossible to achieve any level of consistency across regions without some accepted guidelines to help frame the exception requests and adjudication. 2. Under the current system, the majority of reviewers for any pediatric exceptions request are adult providers. In this context, I fully support the effort but would add the following comments. In response to a prior comment, I believe the proposed 1A exception guidelines regarding children with dilated cardiomyopathy who are 5-10 kg at the time of listing are reasonable. While it is true, some of these children may be VAD eligible, independent of center volume, it is reasonable to request 1A exception under the conditions as proposed. Further, these children are at similar risk and may derive similar benefit as many conventional 1A children with congenital heart disease on milrinone for example. I would however, be a bit more restrictive for this group, and remove "feeding intolerance" from the criteria satisfying the evidence of poor systemic perfusion. The other point I would like to address are the recommendations around children with documented coronary allograft vasculopathy (CAV). The use of revascularization for pediatric CAV has not been shown to significantly alter the natural history of this typically progressive disease. I would propose that a child with CAV of any grade AND graft dysfunction (based on Ejection fraction, PCWP, dysrhythmias perhaps?) as well as any child with CAV2 or higher should be granted a status 1B exception. These children have a real risk of sudden death and would often not be well served as status 2 candidates.

Peter Eckman | 08/27/2020

As an adult transplant cardiologist, I do not believe I am best qualified to comment on pediatric heart transplant exception requests. This patient population is incredibly different from adult candidates, and is asking adult specialists to comment on these candidates is inappropriate. It is clearly in the best interests of pediatric candidates to be evaluated by pediatric heart transplant specialists. I can't imagine a compelling reason to continue the present approach of using unqualified adult heart transplant specialists to perform this essential review task.

Region 5 | 08/28/2020

Region 5 vote: 3 Strongly Support, 20 Support, 10 Neutral/Abstain, 0 Oppose, 0 Strongly Oppose. Region 5 supported the proposal and had the following comments: • The pediatric guidance allows for 1A status for pts on milrinone alone if under 10 kg, we don’t support this and it is contrary to the last revision to listing status • We strongly support the guidance document that has been developed. • We want to thank the committee for their work on this. This guidance addresses many of the gaps presented since the last policy changes for pediatric heart allocation and will certainly be a positive change for families.

Region 7 | 09/10/2020

Region 7 vote: 5 Strongly Support, 8 Support, 4 Neutral/Abstain, 0 Oppose, 0 Strongly Oppose Region 7 supported the proposal and had the following comments: • Attendee stated that these are important changes, and are in support. However, they added that they would like the committee to fully investigate the root causes behind the exception requests, to properly account for those causes as we move toward Continuous Distribution. • An attendee added that they have been advocating for this and believe it is unsafe and unfair to have adult Cardiologists making decisions on behalf of pediatric candidates. This change is overdue and very appropriate. • An attendee asked if they would consider a PRA cutoff like they do in Canada. The Committee encouraged the region to submit more comments like these for the committee to consider.

Region 3 | 09/15/2020

Region 3 vote: 2 Strongly Support; 19 Support; 5 Neutral/Abstain; 1 Oppose, 0 Strongly Oppose: No comments during discussion Comments submitted online during meeting: • This would allow carte blanche status 1B for all Fontan patients • Agree with guidance and not change in policy • Monitor and if guidance does not work we should consider policy change

Ashwin Lal | 09/17/2020

Agree with most of the previous comments particularly from Dr. Rosenthal & Hollander and the move to have a pediatric review panel. 1) I too am concerned about DCM patients between 5-10 kg given 1A status. Experience has increased beyond the highest volume centers and a patient in this age weight range with systemic symptoms would benefit from a VAD. 2) I don't have the same objection to Milrinone therapy threshold. I agree that .5 is not truly high dose but identifies patients who are inotrope dependent. Having this threshold would hopefully avoid the circumstance where someone was deemed inotrope dependent even if they are on a "homeopathic" dose (as a reviewer I have seen adult patients on 2 inotropes where the second inotrope was simply to increase their status). The wording/intent could be clarified. 3) I had a similar question about whether CAV patients need to be admitted to qualify for 1A status. 4) The issue of sensitization is an important one, but agree that there may be too much variability in assessment to put this in writing

Region 8 | 09/22/2020

Region 8 vote: 5 strongly support, 10 support, 6 neutral/abstain, 0 oppose, 0 strongly oppose No comments

American Society of Transplantation | 09/24/2020

The American Society of Transplantation supports this proposal that aims to provide greater clarity and guidance for the status of pediatric candidates for heart transplantation with the following diagnoses: dilated cardiomyopathy, hypertrophic cardiomyopathy, congenital heart disease, cardiac allograft vasculopathy. The proposal makes reasonable recommendations regarding the process to upgrade different types of cardiomyopathy in the pediatric population. Overall, for the majority of types it creates greater alignment with the adult policy. Additional clarity regarding contraindications for mechanical circulatory support would be beneficial in dilated cardiomyopathy candidates. We offer the following points that we believe warrant additional attention before moving this proposal forward for final approval: • Dilated Cardiomyopathy: o Guidance is stratified by weight and inotrope utilization (similar to the adult policy with the absence of specific hemodynamic requirements) o Guidance is also provided for contraindications for mechanical circulatory support. These criteria are somewhat vague – but this does allow for center-specific differences. • Hypertrophic/Restrictive Cardiomyopathy o The proposal suggests this group is most adversely impacted by the current status system. The guidance in this proposal provides a clearer path to upgrade to 1A status and similar to the adult policy. • Single Ventricle Heart Disease o The guidance provides aligns the pediatric policy with the adult policy. • Coronary Allograft Vasculopathy & Re-transplantation o The proposal provides reasonable criteria to guide upgrade to status 1A. • Supporting exceptions in children <10 kg who are not on VAD support. Some of our members disagree with this exception. Their concern is that smaller centers may not be comfortable placing VADs in small children, and this would give children in these centers an advantage over children at larger, more skilled centers. We also feel that this exception could potentially disincentivize centers from placing a VAD and lead to suboptimal outcomes. • Milrinone of 0.5mcg/kg/minutes should not be considered "high dose”. Many centers use this dose for children who are feeding intolerant to improve nutrition. We do not feel that this dose always represents severe illness. • We suggest that the committee consider specifying what the VAD contraindications are for the 5-10kg group. To respond to the request for feedback, we do not think that there are other VAD contraindications that need to be considered at this time. • To respond to the request for feedback, there are not sufficient data to support the use of a stringent “measure of sensitization” for Status 1A listing for CAV candidates.

Anonymous | 09/24/2020

definitely needed as attempts to improve "fairness" to certain groups in allocation policies always seem to cause "unfairness" to other groups. most centers palliate/repair congenital defects and by the time transplant is needed, those pts. have complications/factors that may influence morbidity/survival/longevity. data does not reflect that yet...but given time, it will..... until then, exceptions will need to be implemented & the guide is an excellent start!

Region 1 | 09/24/2020

Region 1 vote: 4 Strongly Support, 3 Support, 5 Neutral/Abstain, 0 Oppose, 0 Strongly Oppose Comments: Region 1 supports this guidance and had no comments.

Region 2 | 09/25/2020

Region 2 vote: 8 Strongly Support, 15 Support, 9 Neutral/Abstain, 0 Oppose, 0 Strongly Oppose Comments: An attendee commented that some of the metrics for inotropic agents and their doses require adjustments to better align with current pediatric pharmacologic management for pediatric patients with end-stage heart disease. What does the committee consider low, medium, and high dosage for this population? They request that the committee spend more time fine-tuning the guidance document to establish more appropriate metrics.

Region 6 | 09/29/2020

Region 6 vote: 9 strongly support; 26 support; 6 neutral/abstain; 0 oppose; 0 strongly oppose. Comments: A member noted that based on past experience with the Pediatric National Liver Review Board that it would be helpful to look into how the guidance document can be translated into an automated process which would help all children rather than just those whose programs are able to craft a good narrative.

Region 10 | 09/29/2020

Region 10 vote: 2 Strongly Support; 10 Support; 13 Neutral/Abstain; 0 Oppose; 0 Strongly Oppose. Comments: No comments

American Society of Transplant Surgeons | 09/29/2020

The American Society of Transplant Surgeons (ASTS) strongly supports the concept of development of a national pediatric review board and a more standardized approach to use of exceptions for pediatric heart transplant patients by developing guidelines; however, we have the following concerns. Regarding the specific questions posed to us and the review of the current guidelines we have the following comments: 1) We would not support having PRA/sensitization play a role in selection of status for pediatric heart transplantation. 2) We support size as the main criteria for special consideration for VAD recipients listed for heart transplantation. We do not support adding any other qualifications at this time. 3) We are concerned the re-transplant focuses on those with coronary allograft vasculopathy (CAV), and ignores those re-transplanted for end-stage restrictive physiology without CAV. We would support clarifying that this latter group could qualify by exceptions recognized for other RCM, such as elevated pulmonary resistance and/or inotrope dependence, or for symptomatic, intractable ascites and pleural effusions. They should be considered for 1B if outpatient and 1A if inpatient.

Society of Pediatric Liver Transplantation | 09/30/2020

The Society of Pediatric Liver Transplantation (SPLIT) supports the Heart Committee’s efforts in creating a guidance document for reviewers on the National Heart Review Board addressing pediatric candidate exception requests. It is crucial that reviewers possess knowledge of pediatric specific diseases in order to make informed decisions regarding the granting or denial of exception scores. We encourage a guidance document to be based on the best available evidence but allow flexibility in unique patient circumstances. We support our pediatric cardiology colleagues in providing the medical details best suited for this guideline

Donna Campbell | 09/30/2020

do not have enough information to comment

NATCO | 09/30/2020

NATCO supports this proposal. This guidance document provides additional information on what reviewers should consider when making these decisions on exception requests for the following diagnoses: dilated cardiomyopathy, hypertrophic cardiomyopathy, congenital heart disease, and cardiac allograft vasculopathy (CAV). There are no contraindications to the use of ventricular assist device (VAD) that should be considered. We support size as the criteria for special consideration for VAD recipients listed for heart transplantation. Milrinone of 0.5mcg/kg/minutes should not be considered "high dose”. We would not support the measurement of sensitization to assist in determining whether CAV candidates should be considered for status 1A listing by exception. We thank the committee for all their efforts on this guidance document which will help with pediatric heart allocation moving forward.

Joseph Hillenburg | 09/30/2020

This guidance document is an important step toward rectifying imbalances within pediatric heart allocation. I would encourage the committee to address the root causes for exceptions, to reduce the number of required exceptions.

Association of Organ Procurement Organizations | 10/01/2020

The Association of Organ Procurement Organizations (AOPO) supports the Heart Transplantation Committee’s guidance regarding the Pediatric Heart Exceptions. Appropriate utilization of exceptions is essential to maximize the gift of donation and maintain public trust in our system of organ allocation.

Transplant Families | 10/01/2020

Transplant Families, the non-profit organization representing pediatric recipient families, patients, and it's leadership, support this document. This guidance document addresses complexities that weren't discussed before in whether the multitude of congenital heart defects vs. cardiomyopathy had priority. There are instances in both groups that could put patients at a higher priority and this is a good start to addressing these. We applaud the committee for instantiating a pediatric review board that understands this group as only they can. We would also encourage the committee to continue to explore root causes for exceptions to reduce number of required exceptions. We thank the committee for their time and continued dedication to the unique concerns of the pediatric population

Pediatric Transplantation Committee | 10/01/2020

The Pediatric Transplantation Committee (the Committee) thanks the Heart Transplantation Committee (Heart Committee) for their efforts in crafting the Guidance Addressing the Use of Pediatric Heart Exceptions and appreciated the opportunity to provide feedback. The Committee generally supports the proposal but provides several points of feedback for the Heart Committee’s consideration. The Committee opposes the inclusion of sensitization to help determine whether a Coronary Allograft Vasculopathy candidate should be considered for Status 1A listing by exception. The Committee also noted that there may be some cases in which single ventricles may not be amenable to Ventricular Assist Devices (VAD) which should be taken under consideration. The Committee urged the Heart Committee to be aware and intentional about striking a balance of equity between single ventricle and cardiomyopathy exception patients. The Committee recommends there be language built into the guidance document to indicate that the document will be updated as needed to accommodate any changes in the process. The Committee sentiment vote was 4 strongly support, 4 support and 1 neutral/abstain for the proposal.

Transplant Coordinators Committee | 10/01/2020

The Transplant Coordinators Committee thanks the Heart Transplantation Committee for the opportunity to comment on the guidance document. The committee noted that guidance for exception requests are extremely helpful. It helps save time, and reduces back and forth with the review boards, being able to see if patients are eligible for exceptions prior to submission as well the necessary data needed. The committee indicated the following sentiment for the guidance document: 5 Strongly Support, 8 Support, 0 Neutral/Abstain, 0 Oppose, 0 Strongly Oppose.

Region 9 | 10/01/2020

Region 9 vote: 4 strongly support, 10 support, 4 abstain/neutral, 0 oppose, 0 strongly oppose. Comments: A member commented that when the policy changed in 2016, it favored congenital patients with higher waitlist mortality and that this is for patients who may not fit neatly into the same category as those on inotropes.

Region 11 | 10/01/2020

Region 11 vote: 5 strongly support, 8 support, 12 neutral/abstain, 0 oppose, 0 strongly oppose. Comments: An attendee provided feedback that centers should be given autonomy to justify their rationale for exception requests. One attendee did not support using PRA to determine status for pediatric heart transplant. There was support from one attendee for using size as an exception criteria for VAD candidates. This attendee did not support adding additional criteria. One attendee expressed concern that the guidance for re-transplant focuses on candidates with coronary allograft vasculopathy (CAV) and does not consider re-transplants for end-stage restrictive physiology without CAV. This attendee went on to comment that they would support clarifying that this latter group could qualify by exceptions recognized for other RCM, such as elevated pulmonary resistance and/or inotrope dependence, or for symptomatic, intractable ascites and pleural effusions and should be considered for 1B if outpatient and 1A if inpatient.

Brian Feingold | 10/01/2020

Thank you for advancing this very important concept and guidance. I have the following specific comments/concerns: 1.With respect to guidance on DCM exceptions, I think this should be very, very restrictive. Otherwise we risk being in the same place we are starting from with regards to DCM candidates being granted exceptions that the data suggest they do not truly need. I think <5kg it is very reasonable to consider exception with guidance as currently is noted. However for 5-10kg and certainly greater should really have higher threshold than what is now noted. The poor cardiac output condition for 5-10kg is really an indication for VAD and so there should be a reason why VAD is potentially contraindicated that is not based on size (akin to the instructions for pts >10kg). 2. For >10kg DCM, as others have commented, life-threatening arrhythmias in typically resolve with VAD and so I do not think this is a reason not to place a VAD. 3. I believe RCM and HCM guidance is reasonable except the arrhythmia clause. Would an ICD not be indicated in such patients and unless ICD/anti-tachycardia pacing is not feasible be a more appropriate indication in such patients? 4. For CHD 1B Fontan exception, I am concerned that we are seeking to rectify this incongruity in the between new adult listing criteria with use of an exception. This seems like a bad idea - it then is incumbent on the center to seek exceptions for all of these patients. Is it still an exception or the rule? What if some centers do not recognize this and their patients who could meet this exception never have it requested? It would seem the appropriate way to fix this incongruity is to revise the adult criteria/allocation such that every pediatric CHD candidate at status 2 is ahead of any adult CHD category 4 candidate. 5. Also for CHD I don't think it is justifiable to have a CHD patient with PLE who is admitted to listing hospital not on inotropes listed status 1A. Plastic bronchitis is more urgently life-threatening typically and this makes sense to me. 5. For ReTx, suggest rephrasing "triple vessel disease" as some patients have severe diffuse disease (would this be triple vessel disease)? Perhaps use ISHLT CAV 3 grade as an alternative to "triple vessel disease"? 6. Finally I think the pediatric heart exception board should have the opportunity to review all exceptions granted and denied every so often (3, 6, 12mo) to reflect on decisions and whether they are truly meeting spirit intended of this guidance. This would also allow for some "institutional memory" for this body so that with volunteer turnover that decision-making does not radically shift (unless intended).

Sam Dey | 10/01/2020

Strongly Support