Liver transplantation for propionic acidemia: Survival and metabolic outcomes
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View abstract on PubMed
Summary
This summary is machine-generated.Liver transplantation (LT) significantly improves outcomes for children with propionic acidemia (PA), a severe metabolic disorder. Living donor liver transplantation effectively reduces metabolic waste and enhances quality of life in pediatric PA patients.
Area Of Science
- Metabolic disorders
- Pediatric surgery
- Organ transplantation
Background
- Propionic acidemia (PA) is a severe inherited metabolic disorder causing multiorgan damage.
- Current management strategies for PA offer limited efficacy.
- Liver transplantation (LT) is a potential treatment, but large-scale evidence is scarce.
Purpose Of The Study
- To evaluate the clinical outcomes of pediatric patients with PA undergoing liver transplantation.
- To assess the effectiveness of living donor liver transplantation in managing PA.
- To analyze the impact of the diagnosis-to-transplant interval on patient outcomes.
Main Methods
- Retrospective analysis of 39 pediatric PA patients who underwent LT from September 2017 to October 2023.
- Inclusion of demographic, surgical, biochemical, and clinical data.
- Comparative analysis based on diagnosis-to-transplant interval (<20 vs. ≥20 months).
Main Results
- 97.4% of patients underwent living donor liver transplantation.
- Significant reductions in key metabolic markers (propionylcarnitine/acetylcarnitine ratio, urinary methylcitrate, 3-hydroxypropionic acid) were observed post-LT.
- Marked improvement in clinical symptoms including motor delay, metabolic acidosis, hyperammonemia, and feeding difficulties (all p<0.001).
- 5-year patient and graft survival rates reached 97.4%.
Conclusions
- LT, particularly living donor liver transplantation, is an effective treatment for pediatric PA.
- Liver transplantation reduces metabolic waste, improves metabolic stability, and enhances quality of life.
- LT is a viable therapeutic option for pediatric patients with metabolic instability due to PA.
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