Liver and/or kidney transplantation in amino and organic acid-related inborn errors of metabolism: An overview on European data.

ABSTRACT:

Background

This study provides a general overview on liver and/or kidney transplantation in patients with an amino and organic acid-related disorder (AOA) with the aim to investigate patient characteristics and global outcome in Europe. This study was an initiative of the E-IMD and the AOA subnetwork of MetabERN.

Methods

A questionnaire was sent to all clinically active European Society for the Study of Inborn Errors of Metabolism (SSIEM) members. The questionnaire focused on transplanted individuals with methylmalonic acidemia (MMA), propionic acidemia (PA), maple syrup urine disease (MSUD), and urea-cycle disorders (UCDs).

Results

We identified 280 transplanted AOA patients (liver transplantation in 20 MMA, 37 PA, 47 MSUD, and 111 UCD patients, kidney or combined li

SUBMITTER: Molema F 

PROVIDER: S-EPMC8247334 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature