Progress in Pediatric Transplantation

In this issue of Pediatrics, Hickner et al1 examined survival progress in pediatric heart, liver, and kidney transplant from 1976 to 2017. The authors found that intent-to-treat survival, waitlist survival, and post-transplant survival improved steadily over the study period for those who received a transplant, regardless of organ. The year the patient was listed for a transplant was a significant predictor of intent-to-treat survival for patients listed for pediatric heart (hazard ratio [HR]: 0.96; P < .001), liver (HR: 0.97; P < .001), and kidney (HR: 0.98; P < .001) transplant.1 These data are encouraging, and this is an excellent analysis. That said, we should not be proud of these findings. Survival differences related to geography and resources remain for children with end-organ disease.Within even the busiest centers pediatric transplant care is low volume; thus, outcomes-based measurement is unreliable, making focused, quality improvement efforts difficult. Even this large analysis suffers from this issue. There is also no robust learning health system to continuously drive improvements in pediatric transplant care in the United States; improvements are mostly related to national secular trends. Action is needed to improve the lives of children with end-organ disease in the United States.First, we must focus on geography variability because this analysis suggests marked geographic heterogeneity in improvement. Regional listing rates per population of children, stratified by social determinants of health, can be used to identify gaps in access. Although, in this article, the authors appropriately take an intention-to-treat approach to waitlist and transplant outcomes, as a parent you just want the best care for your child with end-organ disease. More work is needed to understand the lives of children who never get access to advanced pediatric care that can prevent a transplant or who are never referred for transplant.Second, the physician community should lead progress for children with end-organ disease through a structured national effort. Payers have little leverage to drive clinical change because transplant care is small volume for any individual payer. This is a low priority for health care purchasers, such as employer groups for the same reason. The physician community should organize into an effective learning health system that leverages an engaged, expert physician community, robust data, and shared care pathways to drive iterative quality improvement. Previous experiences of collaborative quality improvement networks can serve as a template for effective approaches to systematically identify regional and center-level variation in pediatric transplant outcomes and offer potential strategies to standardize practice across diverse practice settings, such as evidence-based care pathways, targeted financial incentives, and directed educational interventions.2 The Studies for Pediatric Liver Transplantation group previously demonstrated the feasibility of adapting such a broad-based multi-institutional quality improvement approach to identify high and low performing centers in biliary and arterial complications after pediatric liver transplant and disseminate best practice guidelines to promote high-quality pediatric liver transplant care for patients across all centers. Unfortunately, good work, such as this, has not continued within pediatric transplantation.Third, policy makers must increase their focus on transplant access for children. It is difficult to balance competing interests among transplant stakeholders around organ allocation policy, although progress has been made for children in recent years. It is also difficult to predict how organ allocation policy change will affect children because clinical behavior changes always follow policy changes. New policy efforts should focus on the problem of regional variation in organ donation rates among children.3,4 This analysis notes an interesting trend in Medicaid coverage for transplant patients: the proportion of children who have received transplants with Medicaid as the primary payer has risen rapidly. This may have deleterious implications for pediatric transplant care, and future work should focus on better understanding the implications of this trend. Expanding end-organ disease and transplant programs to serve a primarily Medicaid population will be a financial strain on health systems. Transplant centers must devote large amounts of resources to support underserved patients and families, and this work must be paid for by commercial and public payers through care-coordination billing codes. More researchers must investigate the complex relationship between payment policy and coverage for children with end-organ disease.All children should have access to the best potential outcome, regardless of where they live, who pays for their care, or which transplant center they choose. True progress in pediatric organ transplant should be measured not only by overall improvement in outcomes but also by equitable improvement in outcomes for all patients. Intentional efforts are needed to monitor and eliminate variation in care to ensure equal access to high-quality pediatric organ transplant care for all.