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

Author Department

Surgery

Document Type

Article, Peer-reviewed

Publication Date

12-2025

Abstract

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.

Keywords: Marfan syndrome; heart transplant evaluation; pediatric heart failure; pediatric heart transplantation.

PMID

41243256

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