The management of organic acidaemias: The role of transplantation

This workshop addressed the issue of treatment in propionic acid-aemia (PA) and methylmalonic acidaemia (MMA) and in particular the outcome of convent ional management compared with organ transplantation. Although it appears t hat with medical treatment the mortality for early-onset disease has improv ed, long-term outcome remains poor. However, liver and liver/kidney transpl antation is associated with a high perioperative mortality and long-term co mplications. It offers only a partial cure. The indications for transplanta tion remain unclear. It is recommended that a register of PA and MMA patien ts should be established.