Long-term results of liver transplantation in familial amyloidotic polyneuropathy type I

Background. The aim of this article is to report our experience regarding the survival and the evolution of polyneuropathy of the extremities and autonomic dysfunction in 18 liver transplant patients with familial amyloidotic polyneuropathy type I after a mean follow-up of more than 2.5 years for 13 patients. Methods. The actuarial survival rate of the 18 patients is 72.2% and 60.1%, respectively, at 12 and 58 months. Results. In all the patients we noted clinical improvement of the polyneuropathy of the extremities and autonomic dysfunction during the first 6 months after transplant. The clinical data due to autonomic nervous system involvement showed an earlier improvement than those due to nervous motor involvement. Conclusions. In conclusion, our results suggest that liver transplant may be useful in the treatment of certain cases of familial amyloidotic polyneuropathy to stop the neurological deterioration of the patients and to avoid the fatal end of the disease.