Pediatr Gastroenterol Hepatol Nutr.  2021 May;24(3):288-294. 10.5223/pghn.2021.24.3.288.

Efficacy of Living Donor Liver Transplantation in Patients with Methylmalonic Acidemia

• Jang JG
• Oh SH
• Kim YB
• Kim SH
• Yoo HW
• Lee BH
• Namgoong JM
• Kim DY
• Kim KH
• Song GW
• Moon DB
• Hwang S
• Lee SG
• Kim KM

• KMID: 2515831
• DOI: http://doi.org/10.5223/pghn.2021.24.3.288

Abstract

Purpose
Despite aggressive medical and nutritional management, patients with methylmalonic acidemia (MMA) often suffer from multi-organ damage. Early deceased donor liver transplantation (DDLT) has emerged as an intervention to prevent disease progression. We investigated the efficacy of living donor LT (LDLT) with a potential carrier of MMA and a small volume of graft in patients with MMA as an alternative to DDLT.
Methods
Of five patients (three male, two female; median age 5.7 years; range, 1.3–13.7 years), four underwent carrier LDLT, while one underwent non-carrier auxiliary LDLT. All patients received pre- and post-LT continuous renal replacement therapy and were provided with minimal restriction diet according to serum MMA level after LT. MMA levels in the serum and urine, the incidence of metabolic crisis, and clinical findings before and after LT were compared.
Results
The survival rate was 100% during 2.2 years of follow up period after LT. In all five cases, MMA titer in the serum after transplantation decreased with less restrictive diet. Metabolic crisis was not observed during the follow-up period. In addition, no patient showed progression of severe renal impairment requiring hemodialysis. Progression of delayed cognitive development was not observed. Social functioning with improved neuropsychiatric development was observed.
Conclusion
This study showed that LDLT achieved improved quality of life with less restrictive diet, therefore it could be a feasible alternative option to DDLT for the treatment of patients with MMA, even with an auxiliary LT.

Keyword

Methylmalonic acidemia; Living donors; Liver transplantation; Child