Pediatric Marfan Syndrome and Heart Transplantation: Insights From the PHIS Database

Background Marfan syndrome (MFS) is a connective tissue disorder associated with significant cardiovascular complications, including heart failure. Orthotopic heart transplantation (OHT) is considered controversial in this population due to concerns about post‐transplant aortic complications, particularly in children. Methods We conducted a retrospective review of the Pediatric Health Information System (PHIS) database to identify patients under 18 years of age with a diagnosis of MFS who underwent OHT between 2004 and 2024. Patients were propensity matched (3:1) to non‐MFS OHT recipients. Outcomes included graft failure, aortic events, and re‐transplant‐free survival. Results Ten pediatric MFS patients were identified among 5493 OHT recipients. The median age at OHT was 12.5 years. Over a median follow‐up of 3.73 years, no in‐hospital mortalities or repeat transplants were observed; two patients experienced rejection, and one developed aortic root dilation without requiring intervention. Propensity‐matched analysis showed no significant differences in rejection, aortic events, or transplant‐free survival between MFS and non‐MFS cohorts. Conclusion Though rare, pediatric MFS patients undergoing OHT demonstrated excellent short‐term outcomes with no MFS‐related surgical complications. These findings challenge current exclusionary practices and support further research into long‐term outcomes.