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Multicenter Study
. 2024 Sep;28(6):e14834.
doi: 10.1111/petr.14834.

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

Robert C Minnee  1 , Seisuke Sakamoto  2 , Akinari Fukuda  2 , Hajime Uchida  2 , Kazuya Hirukawa  3 , Masaki Honda  3 , Shinya Okumura  4 , Takashi Ito  4 , Tonguç U Yilmaz  5 , Yitian Fang  1 , Toru Ikegami  6 , Kwang W Lee  7 , Mureo Kasahara  2 ; Vanguard Multi‐Center Study of International Living Donor Liver Transplantation Group
Affiliations
Multicenter Study

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

Robert C Minnee et al. Pediatr Transplant. 2024 Sep.

Abstract

Background: Despite early diagnosis and medical interventions, patients with methylmalonic acidemia (MMA) suffer from multi-organ damage and recurrent metabolic decompensations.

Methods: We 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.

Results: The 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).

Conclusions: LDLT 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.

Keywords: liver transplantation; living donor; methylmalonic acidemia; outcomes; pediatric.

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References

    1. M. Kasahara, R. Horikawa, M. Tagawa, et al., “Current Role of Liver Transplantation for Methylmalonic Acidemia: A Review of the Literature,” Pediatric Transplantation 10, no. 8 (2006): 943–947.
    1. C. Baba, M. Kasahara, Y. Kogure, et al., “Perioperative Management of Living‐Donor Liver Transplantation for Methylmalonic Acidemia,” Paediatric Anaesthesia 26, no. 7 (2016): 694–702.
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    1. P. Forny, F. Horster, D. Ballhausen, et al., “Guidelines for the Diagnosis and Management of Methylmalonic Acidaemia and Propionic Acidaemia: First Revision,” Journal of Inherited Metabolic Disease 44, no. 3 (2021): 566–592.
    1. A. J. Kwong, N. H. Ebel, W. R. Kim, et al., “OPTN/SRTR 2020 Annual Data Report: Liver,” American Journal of Transplantation 22, no. Suppl 2 (2022): 204–309.

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