Orthotopic heart transplantation for hematopoietic stem cell transplantation related end-stage heart failure in pediatric acute monocytic leukemia: a case report

Abstract Introduction Acute monocytic leukemia (AML-M5) is a distinct subtype of acute myeloid leukemia. Hematopoietic stem cell transplantation (HSCT) is a curative treatment option for AML but is associated with various cardiovascular complications. Long-term heart failure after pediatric HSCT is an uncommon cardiovascular complication, with reported incidence rates below 1%. We present a challenging case of end-stage heart failure developing during long-term follow-up after HSCT that was successfully managed with orthotopic heart transplantation. Case report A 15-year-old patient with AML-M5 developed progressive cardiac dysfunction during long-term follow-up after HSCT and ultimately progressed to end-stage heart failure despite guideline-directed medical therapy. Pre-transplantation cardiac function had previously been normal. Management and outcome The patient underwent successful orthotopic heart transplantation. Post-transplantation course was uneventful, and follow-up assessments demonstrated satisfactory graft function with normal ventricular geometry. Histopathological examination of the explanted heart demonstrated pathological changes consistent with advanced dilated cardiomyopathy. Discussion This case highlights the potential for late-onset, progressive heart failure in long-term HSCT survivors. For selected patients with GDMT-refractory end-stage heart failure, orthotopic heart transplantation represents a feasible therapeutic option. Long-term cardiovascular surveillance is essential to facilitate early detection and management of progressive cardiac dysfunction in HSCT recipients.