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Updated: Jul 5, 2025
A Porcine Heterotopic Heart Transplantation Protocol for Delivery of Therapeutics to a Cardiac Allograft
Published on: February 14, 2022
Paediatric heart transplantation: life-saving but not yet a cure
Robert Boucek1, Richard Chinnock2, Janet Scheel3
1Enduring Hearts, Scientific Advisory Committee, Atlanta, GA, USA.
Insights
Pediatric heart transplantation has improved survival rates for infants with hypoplastic left heart syndrome. Further research and funding are crucial to address challenges like wait-list mortality and long-term outcomes.
Area of Science:
- Pediatric Cardiology
- Transplantation Immunology
- Public Health
Background:
- Heart transplantation emerged as a life-saving treatment for infants with hypoplastic left heart syndrome in the 1980s.
- Pediatric heart transplant recipients exhibit immunologic advantages, contributing to improved long-term survival and reduced cardiac allograft vasculopathy.
- Significant advancements in clinical practices have led to approximately 90% of children reaching their first-year post-transplant anniversary.
Purpose of the Study:
- To review the progress and persistent challenges in pediatric heart transplantation.
- To identify key areas for future research and advocacy to improve long-term outcomes for pediatric heart transplant recipients.
- To highlight the disparities in research funding between pediatric and adult heart transplantation.
Main Methods:
- Review of clinical practices and outcomes in pediatric heart transplantation.
- Analysis of factors contributing to survival and graft loss.
- Examination of research funding trends and their impact on pediatric recipients.
Main Results:
- While short-term survival has dramatically improved, long-term survival into adulthood remains a significant challenge.
- High wait-list mortality, particularly in infants, and cardiac allograft vasculopathy are major obstacles.
- Ethnic and racial disparities in transplantation risks require further investigation.
- Research funding disproportionately favors adult recipients, potentially hindering progress in pediatric transplantation.
Conclusions:
- Continued research and advocacy are essential to overcome current limitations in pediatric heart transplantation.
- Addressing wait-list mortality, graft loss, and ethnic disparities is critical for improving long-term outcomes.
- Equitable funding and policy incentives, like Orphan Drug rules, are vital to advance pediatric heart transplant care worldwide.
Abstract:
In the 1980s, heart transplantation was the first successful treatment for infants born with hypoplastic left heart syndrome. Infants who have required heart transplantation benefit from immunologic "advantages," including long-term survival free from cardiac allograft vasculopathy. Currently ∼ 90% of children undergoing a heart transplant are reaching their first-year anniversary and the clinical practices of paediatric heart transplantation have dramatically improved. These successes are largely attributed to research sponsored by the Pediatric Heart Transplant Study Group, the International Society of Heart and Lung Transplantation and, more recently, the Non-profits Enduring Hearts and Additional Ventures. Despite these successes, the field is challenged to increase progress to achieve long-term survival into adulthood. The wait-list mortality, especially among infants, is unacceptably high often leading to palliative measures that can increase post-transplant mortality. Cardiac allograft vasculopathy remains a major cause for progressive graft loss of function and sudden death. The relative tolerance seen in immature recipients has not been translated to modifying older recipients' post-transplant outcomes. The modifiable cause(s) for the increased risks of transplantation in children of different ethnicities and races require definition. Addressing these challenges faces the reality that for-profit research favours funding adult recipients, with ∼ 10-fold greater numbers, and their more modest longevity goals. Advocacy for funding "incentives" such as the Orphan Drug rules in the United States and upholding principles of equity and inclusion are critical to addressing the challenges of paediatric heart transplant recipients worldwide.
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