Which Kids Are ‘Too Sick’ for Solid Organ Transplant?

By Amy Bobrowski, MD

At the annual meeting of the American Transplant Congress (ATC) in June 2023, experts from all fields of organ transplantation gathered to discuss advances and challenges that continue to emerge as these areas advance. I was privileged to moderate one of the “Meet the Expert” sessions entitled Ethical Issues in Pediatric Transplantation: Which Kids are “Too Sick” for Solid Organ Transplant?

Participating in this discussion were pediatric transplant surgeons, pediatric transplant nephrologists/cardiologists/hepatologists, transplant infectious disease specialists, and transplant pharmacists. Discussion was largely led by pediatric nephrologist and medical ethicist, Dr. Aviva Goldberg of the University of Manitoba in Winnipeg, Canada.

When the decision is not straightforward

As pediatric transplant professionals, we often care for children with end organ disease associated with or complicated by rare, genetic, multisystem or neurodevelopmental comorbidities; these can be fixed or progressive. As medical and transplant sciences advance and patients with these conditions survive longer into adulthood, it is becoming more complicated to determine what is in the best interest of some of these patients and their families.

When it comes to decisions about whether or how to proceed with transplantation, issues of quality of life and a balance of risk versus benefit can be complicated and value-laden. While improving transplant outcomes make moving forward with transplantation the best option for the majority of children with end stage organ failure, some of these unique patients may experience an increased likelihood of complications and side effects from the transplant surgery and immunosuppressive medications. This may in turn adversely affect the long-term survival and quality-of-life benefits typically seen in the more “standard” pediatric organ transplant recipient.

There are no clear universal guidelines for determining transplant candidacy in these situations, and the decision of whether to list the same patient for an organ may vary from institute to institute, depending on a particular transplant center’s prior experience with a given diagnosis or situation.

A proposed framework

A framework for discussing such cases, both between the medical and surgical teams and with the patient and their family, was put forward. The first step is to clarify the question. For a given organ failure diagnosis in a particular patient, the question is not whether to provide treatment. It is whether to treat with medical management versus transplantation. Both options may best integrate the involvement of a palliative care team, as transplantation does not guarantee any cure and is often still associated with markedly decreased life expectancy, especially when combined with certain comorbidities.

The personal experiences and desires of the families caring for these children also need to be taken into account. For example, how is the family’s quality of life, home life and burden of care affected by the different treatment options being considered? These expectations, however, also need to be put into the perspective of what the patient’s life will realistically be like after a successful transplant procedure, with or without possible associated complications. This is part of the process of creating “shared expectations” between the medical team and the patient’s family prior to making any decisions about moving forward with transplantation.

Continued challenges and next steps

Unfortunately, small patient numbers mean that meaningful transplant outcome data is not yet available to represent the exact individual situation in many of these cases, and therefore limits truly evidence-based decision-making. We as transplant professionals are sometimes tasked with reaching out to our local, regional, national and international communities for anecdotal experience with which to guide our recommendations (which may include referral to a center with more experience with a given rare diagnosis, if appropriate). We also postulate how the effects of organ transplantation, as well as its associated medications and possible complications, will affect the usual natural history of the underlying condition. An example of this would be considering how significantly the risk of respiratory infection in a non-ambulatory patient with underlying lung disease may be increased with chronic immunosuppressive medications, and weighing this against how much a transplant may or may not increase their life span and/or quality of remaining life.

These questions are not easy to answer, and there is not always one correct answer. The path finally determined should be highly patient- and family-specific. Finding an expert multidisciplinary team that will take the time to thoroughly explore these details and discuss them in a manner understandable to families is crucial as we continue to move the dial toward offering the lifesaving and life-prolonging benefits of organ transplantation to more and more chronically ill children.

About the author: Dr. Bobrowski is Section Head of the Center for Pediatric Nephrology and Hypertension and Medical Director of Pediatric Kidney Transplantation.