Research for improvement of care for pediatric heart transplant patients

In the past nearly-six decades since the first pediatric heart transplant, the field has made great strides. Yet, there remains substantial room for improvement. Wait time, graft longevity, cardiac allograft vasculopathy, rejection monitoring, and balancing immunosuppression with side effects remain significant challenges and affect patient quality of life. We review milestones in pediatric heart transplant research, including the formation of transplant registries, major multicenter prospective studies, and the first randomized clinical trial in pediatric heart transplantation. We discuss ongoing challenges to research in this field, including small population size, heterogeneity, and difficulty with identifying appropriate endpoints. We examine historical regulatory barriers and how they have been addressed to date. Future directions, including key areas of focus and funding pathways are also discussed.