Long‐Term Outcomes of Living Donor Liver Transplantation for Methylmalonic Acidemia

Abstract

ABSTRACTBackgroundDespite early diagnosis and medical interventions, patients with methylmalonic acidemia (MMA) suffer from multi‐organ damage and recurrent metabolic decompensations.MethodsWe conducted the largest retrospective multi‐center cohort study so far, involving five transplant centers (NCCHD, KUH, KUHP, ATAK, and EMC), and identified all MMA patients (n = 38) undergoing LDLT in the past two decades. Our primary outcome was patient survival, and secondary outcomes included death‐censored graft survival and posttransplant complications.ResultsThe overall 10‐year patient survival and death‐censored graft survival rates were 92% and 97%, respectively. Patients who underwent LDLT within 2 years of MMA onset showed significantly higher 10‐year patient survival compared to those with an interval more than 2 years (100% vs. 81%, p = 0.038), although the death‐censored graft survival were not statistically different (100% vs. 93%, p = 0.22). Over the long‐term follow‐up, 14 patients (37%) experienced intellectual disability, while two patients developed neurological complications, three patients experienced renal dysfunction, and one patient had biliary anastomotic stricture. The MMA level significantly decreased from 2218.5 mmol/L preoperative to 307.5 mmol/L postoperative (p = 0.038).ConclusionsLDLT achieves favorable long‐term patient and graft survival outcomes for MMA patients. While not resulting in complete cure, our findings support the consideration of early LDLT within 2 years of disease onset. This approach holds the potential to mitigate recurrent metabolic decompensations, and preserve the long‐term renal function.