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 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.