Cost‐effectiveness of liver transplantation in methylmalonic and propionic acidemias
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Meng Li, Andre Dick, Martin Montenovo, Simon Horslen, Ryan Hansen – 19 May 2015 – Propionic acidemia (PA) and classical methylmalonic acidemia (MMA) are rare inborn errors of metabolism that can cause early mortality and significant morbidity. The mainstay of disease management is lifelong protein restriction. As an alternative, liver transplantation (LT) may improve survival, quality of life, and prevent further neurological deterioration. The aim of our study was to estimate the incremental costs and outcomes of LT versus nutritional support in patients with early‐onset MMA or PA.