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The management of organic acidaemias: The role of transplantation
Author(s) -
Leonard J. V.,
Walter J. H.,
McKiernan P. J.
Publication year - 2001
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1023/a:1010395724012
Subject(s) - medicine , transplantation , liver transplantation , intensive care medicine , perioperative , kidney transplantation , disease , surgery
This workshop addressed the issue of treatment in propionic acid‐aemia (PA) and methylmalonic acidaemia (MMA) and in particular the outcome of conventional management compared with organ transplantation. Although it appears that with medical treatment the mortality for early‐onset disease has improved, long‐term outcome remains poor. However, liver and liver/kidney transplantation is associated with a high perioperative mortality and long‐term complications. It offers only a partial cure. The indications for transplantation remain unclear. It is recommended that a register of PA and MMA patients should be established.