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Author Spotlight: Evaluating Therapeutic Strategies to Enhance Liver Regeneration
Published on: May 24, 2024
Efficacy of Living Donor Liver Transplantation in Patients with Methylmalonic Acidemia
Jae Guk Jang1, Seak Hee Oh1, Yu Bin Kim1
1Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
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.
Insights
Living donor liver transplantation (LDLT) offers a promising alternative for methylmalonic acidemia (MMA) patients, improving quality of life with less dietary restriction and preventing metabolic crises.
Area of Science:
- Hepatology
- Metabolic Disorders
- Transplantation Immunology
Background:
- Methylmalonic acidemia (MMA) is a rare genetic disorder leading to multi-organ damage despite medical management.
- Early deceased donor liver transplantation (DDLT) is used to halt MMA progression.
- Alternative strategies are needed for MMA patients awaiting DDLT.
Purpose of the Study:
- To evaluate the efficacy of living donor liver transplantation (LDLT) in patients with MMA.
- To assess LDLT as an alternative to DDLT, considering potential carriers and small graft volumes.
- To determine if LDLT can improve quality of life and reduce dietary restrictions for MMA patients.
Main Methods:
- Five MMA patients underwent LDLT (four carrier, one auxiliary non-carrier).
- Continuous renal replacement therapy was administered pre- and post-transplant.
- Serum and urine MMA levels, metabolic crisis incidence, and clinical outcomes were compared pre- and post-LT.
Main Results:
- 100% survival rate observed over a 2.2-year follow-up period.
- Post-transplant MMA levels decreased, allowing for a less restrictive diet.
- No metabolic crises, progression of renal impairment, or cognitive delay were observed; improved social functioning noted.
Conclusions:
- LDLT is a feasible treatment for MMA, offering an improved quality of life.
- LDLT enables less restrictive dietary management for MMA patients.
- Auxiliary LDLT is a viable option for MMA treatment.
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