Ethical principles of pediatric organ allocation
Updated November 2014
The purpose of this collaboration between the OPTN Pediatric Transplantation and Ethics Committees is to provide guidance about how organ allocation policies should address the needs of pediatric patients within the ethical framework established by the National Organ Transplant Act and the Final Rule from US Department of Health and Human Services. The National Organ Transplant Act charges the OPTN to "recognize the differences in health and in organ transplantation issues between children and adults throughout the system and adopt criteria, policies, and procedures that address the unique health care needs of children."1 The Final Rule requires that the OPTN develop policies for the "equitable allocation" of deceased donor organs, while also requiring that these policies achieve "best use" of deceased donor organs.2 In June 2010, the Ethics Committee published "Ethical principles in the allocation of human organs," which affirmed that the principles of justice and utility must be balanced in organ allocation.3
In this new analysis, we describe justifications for pediatric priority in organ allocation by focusing primarily on four areas, the Prudential Lifespan Account, the Fair Innings Argument, the "Maximin" Principle, and the concept of utility. The Committees recognize that the arguments for pediatric priority in organ allocation must be considered within the clinical context of each type of organ transplantation and the alternative therapies available while waiting for an organ.
The Committees are aware that many stakeholders in transplantation feel particularly sympathetic to the needs of children. Providing resources to protect children and help them thrive is a fundamental human instinct.4 This conviction has been articulated by the international community. For example, in 1959, the United Nations General Assembly first adopted the Declaration of the Rights of the Child, which still applies and states:
Whereas the child, by reason of his physical and mental immaturity, needs special safeguards and care, including legal protection ... Whereas mankind owes to the child the best it has to give ... The child shall enjoy special protection, and shall be given opportunities and facilities, by law and by other means, to enable him to develop physically, mentally, morally, spiritually and socially in a healthy and normal manner ... In the enactment of laws for this purpose, the best interests of the child shall be the paramount consideration.5
III. The Prudential Lifespan Account
The prudent allocation of healthcare resources over the lifespan
In addition to the survival and quality of life benefits enjoyed by transplant recipients at any age, children with end-stage organ failure have a time-limited opportunity for growth and development and may suffer lifelong consequences if not expeditiously transplanted. As a result, these pediatric candidates have the potential to receive unique benefits from transplantation that will positively affect their lives as children and later, as adults. An allocation system established to promote this set of unique benefits for pediatric patients could be supported by the Prudential Lifespan Account.
Bioethicist Norman Daniels, in his Prudential Lifespan Account, argues that we should make decisions about resource allocation in society similar to how an individual would if he or she did not know his or her exact age (or life expectancy) but had to make decisions about allocation across the stages of his or her own individual life. Daniels explains:
If we are concerned with net benefits within a life, we can appeal to a standard principle of rational choice: It is rational and prudent that a person take from one stage of his life to give to another in order to improve his life as a whole.6
Instead of viewing different age groups as competing for scarce resources, such as deceased donor organs, the Prudential Lifespan Account challenges us to come to a consensus in how each individual would want to invest resources across one life with the goal to “make a life go as well as possible.”6 This consensus can be reached by imagining that each person must make allocation decisions for his or her own life while blinded as to his or her own age. The Prudential Lifespan Account draws attention to the universality of age, making this characteristic fundamentally different from other demographics such as race. From this perspective, as a society, it makes sense to preferentially allocate resources to children and young people in order to maximize the potential for these individuals to thrive in early and later stages of life.7
Before we can determine how we would want resources distributed throughout our lives, the Prudential Lifespan Account requires that we first develop a way to achieve equitable resource allocation among all individuals in society. A complete scheme of equitable allocation addresses both interpersonal issues of distributive justice, as well as distribution across one’s whole life. The “Ethical Principles to be Considered in the Allocation of Human Organs” document provides important guidance.3 We affirm the core distributive requirements of this document, specifically the prohibition against discrimination in the allocation system based on race, gender, socioeconomic group, or social usefulness. “In a public program, all members of the public are morally entitled to fair access to its benefits.”3 Furthermore “even if data were to show that socially disadvantaged groups have worse transplant outcomes, considerations of justice require that patients be assessed individually rather than only by group membership in an attempt to reduce healthcare disparities related to social inequities.”3 Within the broader ethical framework for organ allocation, the Prudential Lifespan Account provides useful guidance about the prioritization of children.
The Prudential Lifespan Account provides a reasonable basis to prioritize children with End Stage Renal Disease (ESRD) for kidney transplantation, rather than subjecting them to a prolonged period of dialysis. Despite the relative rarity of ESRD in children, its long-term consequences are fairly well-documented in the medical literature and include cognitive delays in fine and gross motor, language, learning, and psychosocial development, as well as growth impairment.8 Early age of onset of ESRD and longer duration may accelerate the development of comorbidities, such as cardiovascular disease.9 Kidney transplant corrects, where dialysis only mitigates, the harmful effects on the maturing brain of several conditions associated with ESRD, including uremia and malnutrition.10,11,12 Early transplantation affords the pediatric candidate a better quality of life in childhood, including regular school attendance. Transplant prior to marked growth impairment can prevent lifelong growth deficits and delayed puberty.13,14,15,16 Given these time-limited opportunities to benefit from transplant, it is prudent to prioritize allocation for kidney in childhood versus adulthood to remedy the unique problems of ESRD in childhood. These human capital gains are likely to improve quality of life and in some cases, may be expected to prolong life.
IV. Fair Innings
The “Fair Innings” perspective on equity also provides a relevant basis for pediatric priority in organ allocation. The Fair Innings perspective argues that every individual deserves to experience a full life and that the allocation of society’s resources should try to maximize the opportunity for each person to reach a full lifespan. Children with end-stage organ failure are at risk of premature death, which denies them opportunities in adulthood to complete their education, establish a career, or have a family.19
V. The "Maximin" Principle
Maximizing the minimum benefit to the least advantaged or giving priority to the most disadvantaged groups
When allocating a scarce resource, such as deceased donor organs, inequalities will always exist. There is a strong ethical argument that society should only tolerate inequality in our organ allocation system when those inequalities are arranged so that “they are the greatest benefit to the least-advantaged members of society” (philosopher John Rawls).20 While it is sometimes difficult to achieve consensus on who among those waiting for a lifesaving transplant is most disadvantaged, there are several factors that make pediatric candidates particularly vulnerable and in need of expeditious transplant. The particular disadvantages faced by children with end-stage organ failure include diminished quality of life during development, age and size-specific barriers to transplant, unique challenges in providing life-sustaining therapy for pediatric patients awaiting transplant, and the risk of premature death.
Children with end-stage organ failure suffer a diminished quality of life in childhood, the effects of which may extend into adulthood. As previously discussed, children have a time-limited opportunity for growth and development, and end-stage organ failure in childhood is associated with cognitive delays, growth deficits, delayed puberty, and comorbidities. Absence from school can have a negative impact on social integration, academic achievement, and future employment opportunities. Social isolation results from prolonged absence from school and exclusion from normal activity with family and friends. Children experiencing such isolation often have poor self-esteem and are at risk of suffering from anxiety or depression.21 Healthy childhood confers a lifelong advantage that children in need of organ transplant do not have.
Pediatric candidates also experience barriers to transplantation as a result of their small size and developing anatomy. In addition to the universal issue of donor scarcity, availability of organs is further restricted to pediatric patients requiring size-matched organs. For this reason, children on the waiting list may need to have ready access to a particular subset of organs for which anatomical compatibility will allow transplantation.
The lack of availability of life-sustaining therapies while awaiting transplant further compounds the problem of donor scarcity for pediatric candidates. Technologies to manage end-stage organ failure while waiting for an appropriate organ, also known as Bridge to Transplant technologies, for pediatric patients are limited with inconclusive evidence of success. Bridge therapy for pediatric heart candidates have significant complications, which increase with the duration of technological support and can require intensive multi-disciplinary rehabilitation.22
This had led some researchers, such as Fiser, et al, to insist that alternative therapies “must be developed in the pediatric population that will allow for improved outcomes, comparable with outcomes achieved in the adult population.”23 Children on some forms of mechanical circulatory support have increased risk of stroke compared to adults and are unlikely to be discharged home.24 Mechanical circulatory support options are very limited for infants and children weighing less than 5 kilograms and have a suboptimal success rate.24 Organ scarcity and unavailability of life-sustaining therapy contribute to high waitlist mortality rates, especially among the smallest pediatric candidates (less than 6 years old) waiting for any organ and all pediatric lung candidates.
Even the long-term modality of dialysis for pediatric ESRD has limitations for providing high quality care for children awaiting transplantation and must be considered. Vascular access for chronic dialysis may be difficult to place due to small blood vessels.25 In young children with arteriovenous fistula/graft access, the insertion of large bore needles with every treatment is often difficult for them, their families, and dialysis staff. Dialysis units experienced in providing care for children are often far from the child’s home and school.
Children with end-organ failure are at risk of premature death, which denies them opportunities to complete their education, establish a career, or have a family. The earlier in life a person receives an organ transplant, the more likely that he or she will be able to lead a productive and fulfilling life.26
VI. Utility Considerations
In organ transplantation, utility outcomes are typically measured in terms of patient or allograft survival. The Committees recognize that preferentially allocating organs to children will be consistent with the goal of maximizing utility in some but not all circumstances. Yet, across the entire population of pediatric versus adult transplant recipients, pediatric transplant recipients will on average enjoy lower mortality rates due to the strong association between younger age and longer survival.
As an example, in kidney transplantation, some subgroups of children pose particular surgical challenges for transplantation (e.g. children under 2 years or those with complicated urological anomalies) or have high rates of organ rejection vs. adults (e.g. adolescents). One study of recipients of highest-quality deceased donor kidneys demonstrated that adolescents had allograft survival that was lower than most other age groups. Mortality rates, however, were lower in all pediatric age groups than among adult recipients over age 40 years.27
Overall patient survival is greater for pediatric recipients than adults. We analyzed OPTN data for ten and twenty year Kaplan-Meier patient survival of solitary deceased donor heart, lung, liver, and kidney transplants performed from 1990 to 1997. While 10 year patient survival rates among children less than 18 years old at transplant is only slightly higher than that of adult recipients of heart and lung transplants, pediatric liver and kidney recipients had a markedly better 10 year patient survival than adult recipients (74% vs. 56% and 90% vs. 65%, respectively). For recipients of any organ, children less than 18 years old have over two times the 20 year patient survival rate of adults.
Drawing from regulatory guidance and ethical principles, we find that there is a reasonable basis for giving preference to pediatric transplant candidates for allocation. This preferential allocation must take into account the organ-specific clinical context faced by candidates of all ages.
1. National Organ Transplantation Act (NOTA), 42 USC §273 et seq.
2. Organ Procurement and Transplantation Network Final Rule, 42 CFR 121 et seq. URL: http://ecfr.gpoaccess.gov/cgi/t/text/textidx?c=ecfr&tpl=/ecfrbrowse/Title42/42cfr121_main_02.tpl.
3. "Ethical Principles in the Allocation of Human Organs," Organ Procurement and Transplantation Network, accessed September 30, 2014, http://optn.transplant.hrsa.gov/resources/ethics.
4. Erikson, E.H. Childhood & Society. New York: WW Norton & Co., 1950.
5. Declaration of the Rights of the Child. 1386 (XIV) Item 64. United Nations General Assembly. In; 20 November 1959.
6. Daniels, N. Just Health: Meeting Health Needs Fairly. New York: Cambridge University Press, 2008.
7. Daniels, N. "Global Aging and the Allocation of Health Care Across the Life Span." The American Journal of Bioethics 13 (2013): 1-2.
8. Hooper, S.R., A.C. Gerson, R.W. Butler, et al. "Neurocognitive functioning of children and adolescents with mild-to-moderate chronic kidney disease." Clin J Am Soc Nephrol 6 (2011): 1824-30.
9. Parekh, R.S., C.E. Carroll, R.A. Wolfe, and F.K. Port. "Cardiovascular mortality in children and young adults with end-stage kidney disease." The Journal of Pediatrics 141 (2002): 191-7.
10. Davis, I.D., P. Chang, and T.E. Nevins. "Successful Renal Transplantation Accelerates Development in Young Uremic Children." Pediatrics 86 (1990): 594-600.
11. Mendley, S.R., M.B. Matheson, S. Shinnar, et al. "Duration of chronic kidney disease reduces attention and executive function in pediatric patients." International Society of Nephrology (2014), accessed September 30, 2014, doi: 10.1038/ki.2014.323.
12. Icard, P., S.R. Hooper, D.S. Gipson, and M.E. de Ferris. "Congnitive Improvement in Children with CKD after Transplant." Pediatric Transplant 14 (2010): 887-90.
13. Ellis, D. "Growth and renal function after steroid-free tacrolimus-based immunosuppression in children with renal transplants." Pediatric Nephrology 14 (2000): 689-94.
14. Sarwal, M.M., R.B. Ettenger, V. Dharnidharka, et al. "Complete Steroid Avoidance is Effective and Safe in Children with Renal Transplants: A Multicenter Randomized Trial with Three-Year Follow-Up." American Journal of Transplantation 12 (2012): 2719-29.
15. Wittenhagen, P., H.C. Thiessen, F. Baudier, et al. "Long-term experience of steroid-free pediatric renal transplantation: Effects on graft function, body mass index, and longitudinal growth." Pediatric Transplantation 18 (2014): 35-41.
16. Herthelius, M., G. Celsi, S. Edström Halling, et al. "Renal transplantation in infants and small children." Pediatric Nephrology 27 (2012): 145-150.
17. Mohammed, S., A. Grimberg, E. Rand, et al. "Long-Term Linear Growth and Puberty in Pediatric Liver Transplant Recipients." The Journal of Pediatrics 163 (2013): 1354-60.
18. Knecht, K.R. and W.R. Morrow, "Growth Following Pediatric Heart Transplantation," in Handbook of Growth and Growth Monitoring in Health and Disease: Volume 1, ed. Victor R. Preedy (New York: Springer, 2012), 1841-51.
19. Williams, A., "Intergenerational Equity: An Exploration of the 'Fair Innings' Argument." Health Economics 6 (1997): 117-32.
20. Rawls, J. A Theory of Justice. Cambridge: Belknap Press, 1971.
21. Anthony, S.J., S. Pollock BarZiv, and V.L Ng. "Quality of Life After Pediatric Solid Organ Transplantation." Pediatric Clinics of North America 57 (2010): 559-74.
22. Whitmore, P., S. Nelligan, A. Goldman, M.E. Jones, and M. Burch. "Resource Implications of ECMO as a Bridge to Pediatric Heart Transplant." Abstract 173 in The Journal of Heart and Lung Transplantation S130 (2003).
23. Fiser, W.P., A.T. Yetman, R.J. Gunselman, et al. "Pediatric Arteriovenous Extracorporeal Membrane Oxygenation (ECMO) as a Bridge to Cardiac Transplantation." The Journal of Heart and Lung Transplantation 22 (2003): 770-7.
24. Almond, C.S., D.L. Morales, E.H. Blackstone, et al. "Berlin Heart EXCOR Pediatric Ventricular Assist Device for Bridge to Heart Transplantation in US Children." Circulation 127 (2013): 1702-11.
25. Chand, D.H., D. Geary, H. Patel, et al. Barriers, biases, and beliefs about arteriovenous fistula placement in children: A survey of the International Pediatric Fistula First Initiative (IPFFI) within the Midwest Pediatric Nephrology Consortium (MWPNC). Hemodialysis international International Symposium on Home Hemodialysis 2014.
26. Kamm, F. Morality, Mortality: Volume 1: Death and Whom to Save from It. New York: Oxford University Press, 1993.
27. Levine MH, Reese PP, Wood A, et al. "Inferior Allograft Outcomes in Adolescent Recipients of Renal Transplants from Ideal Deceased Donors." Ann Surg 255 (2012) 556-64.