Early liver transplantation improves familial amyloidotic polyneuropathy patients' survival

Since the first liver transplantation for familial amyloidotic polyneuropathy was performed in 1990, it has become an accepted treatment for this systemic amyloid disorder. Liver transplantation halts the production of the mutated amyloidogenic transthyretin, and thereby amyloid formation, and also progression of the majority of symptoms. Improvement in survival from onset of disease in transplanted patients compared to non-transplanted subjects has of yet not been demonstrated, partly because of the natural relatively slow progression of the disease with an expected median survival of 13 years. In this retrospective study we compared the early initial series (n = 34) of transplantations, where severely malnourished patients were accepted, with a later series (n = 27) of transplants, as well as a control group (n = 19) consisting of non-transplanted patients. For transplanted patients with an modified body mass index (mBMI) above 600 an improved survival was noted compared with that of non-transplanted historical controls. So far no difference in survival between the early and late series has been found. Our previous recommendation of selection of patients primarily according to their nutritional status appears to be well justified, since it is now possible to demonstrate an increased survival for the transplanted group of patients with a preserved nutritional status (mBMI > 600) compared to the control group of non-transplanted patients.