Background: Familial amyloidosis polyneuropathy (FAP) is a rare, progressive, and life-threatening disease inherited in the autosomal dominant pattern. Liver transplantation is the only proven disease-modifying treatment to date.
Aim: To study the long-term outcomes of patients transplanted for FAP under a multidisciplinary team care.
Methods: We included adult patients who were transplanted for FAP indication and were followed up in a relevant clinic or admitted in our department.
Results: Twelve patients (6 male) with a mean age of 43 years and mean follow-up post-transplant of 100 months were included. Three patients died in this period, 1 due to a disease-related cause. All patients had peripheral neuropathy (25% severe). Eighty-three percent had autonomic nervous system dysfunction; all men, except one, erectile dysfunction; and half of the patients several genitourinary manifestations. Gastrointestinal involvement was present in 75% of the patients. The severity of several complications related to FAP was found to be associated with waiting on the transplant list for more than 12 months.
Conclusions: Patients transplanted for FAP have a long survival. Prolonged stay on the transplant waiting list is associated with frequency and severity of disease complications. These patients are best managed in the context of multidisciplinary team care.
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