Liver transplantation is the only effective treatment of familial amyloidotic polyneuropathy type I (FAP). The aim of the present investigation was to identify factors at the time of submission for transplantation that had impact on survival, with special reference to gastrointestinal disturbances. All 28 liver-transplanted FAP patients evaluated at Umeå University Hospital were included in the study. A modified body mass index was used to assess nutritional status. Intestinal examinations were performed to diagnose bile acid malabsorption, gastric retention, and bacterial contamination of the small bowel. A significantly improved survival rate was found for patients in a good nutritional state (
P=0.002). Peripheral neurological symptoms were unrelated to survival, whereas increased mortality was found for patients with bile acid malabsorption (
P<0.05). bacterial="" contamination="" and="" gastric="" retention="" were="" common="" complications="" of="" the="" disease.="" in="" conclusion,="" malabsorption="" and="" malnutrition="" have="" a="" profound="" impact="" on="" the="" outcome="" of="" liver="" transplantation="" for="" familial="" amyloidotic="">
Key words hereditary amyloidosis - inborn errors of metabolism - malabsorption - fat - bile acids - liver transplantation
The study was supported by grants from the patients association FAMY, Umeå Health District, the Swedish Medical Research Council, grant 19X-11240, Umeå University and the Joint Committee of the Northern Health Districts.