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Liver Transplantation for Propionic Acidemia: A Multicenter-linked Database Analysis
Sophoclis P Alexopoulos1, Lea Matsuoka1, Einar Hafberg2
1Division of Hepatobiliary Surgery and Liver Transplantation.
Insights
Liver transplantation (LT) is a viable treatment for propionic acidemia (PA), a rare metabolic disorder. This study found acceptable graft and patient survival rates for PA patients undergoing LT, with similar outcomes to non-PA recipients.
Area of Science:
- Metabolic disorders
- Inborn errors of metabolism
- Genetics and genomics
Background:
- Propionic acidemia (PA) is a rare genetic disorder affecting amino acid and fatty acid metabolism.
- PA often leads to severe neurological damage despite medical management.
- Liver transplantation (LT) is explored as a cellular therapy for PA, but data is limited.
Purpose of the Study:
- To evaluate the efficacy and outcomes of liver transplantation (LT) in pediatric patients with propionic acidemia (PA).
- To analyze the largest available cohort of PA patients who have undergone LT.
Main Methods:
- A multicenter, retrospective analysis of the Scientific Registry of Transplant Recipients and Pediatric Health Information System databases.
- Included pediatric LT recipients over a 16-year period, with a focus on 23 PA patients.
Main Results:
- The 1-, 3-, and 5-year graft survival rates were 84.6%, and patient survival rates were 89.5%.
- No significant difference in graft or patient survival was observed between PA and non-PA LT recipients.
- PA LT recipients had a higher postoperative readmission rate (90.5% vs 72.8%).
Conclusions:
- Liver transplantation is a viable treatment option for children diagnosed with propionic acidemia.
- LT offers acceptable graft and patient survival outcomes for PA, comparable to other pediatric LT recipients.
Objectives:
Propionic acidemia (PA) is a rare inborn error of metabolism resulting from deficiency in the enzyme necessary for catabolism of branched-chain amino acids, some odd chain fatty acids and cholesterol. Despite optimal medical management, PA often leads to acute and progressive neurological injury. Reports on liver transplantation (LT) as a cellular therapy are limited and varied. The objective of this study was to examine the largest collection of patients who underwent LT for PA.
Methods:
Examining the Scientific Registry of Transplant Recipients and the Pediatric Health Information System administrative billing databases, we performed a multicenter, retrospective analysis of LT over a 16-year period. During this period, 4849 pediatric LT were performed out of which 23 were done for PA at 10 different centers.
Results:
The majority of recipients were 5 years of age or younger and had status 1b exception points at the time of transplant. The 1-, 3-, and 5-year graft survival for PA LT recipients was 84.6% and the 1-, 3, and 5-year patient survival was 89.5%. There was no significant difference in graft or patient survival between PA and non-PA LT recipients. Despite historical data to the contrary, we did not find an increased incidence of hepatic arterial thrombosis in patients undergoing LT for PA. Patients in the PA LT group, however, had a significantly higher postoperative rate of readmission compared with the non-PA LT group (90.5% vs 72.8%, P = 0.021).
Conclusion:
LT for children with PA is a viable treatment option with acceptable outcomes.
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