Ten years of international experience with liver transplantation for familial amyloidotic polyneuropathy: results from the familial amyloidotic polyneuropathy world transplant registry

Abstract

We believe that the FAPWTR has become a valuable tool that will help to accurately evaluate the potential risks and benefits of OLT in patients with FAP and promote a fruitful collaboration between centers engaged in this field.

Disease‐associated prion protein is not detectable in human systemic amyloid deposits

“…Thousands of patients with systemic amyloidosis worldwide have been on chronic haemodialysis for end-stage renal failure, and there have been over 200 domino transplants of livers from patients with transthyretin amyloidosis into recipients with terminal liver failure without any sign of prion disease transmission [9,10]. Similarly, systemic amyloidosis has not been reported in patients with prion disease and, although PrP Sc may be found in the peripheral nervous system and elements of the lymphoreticular system [11,12], careful study of cases of vCJD has found no PrP res in the heart [13].…”

Ten Years of Experience with Liver Transplantation for Familial Amyloid Polyneuropathy in Japan: Outcomes of Living Donor Liver Transplantations

“…FAPWTR reported that the type of TTR gene mutation is a useful prognostic parameter (7,15). A better 5-year patient survival was reported in Val30Met TTR type FAP patients, as compared to those with non-Val30Met TTR type FAP (80% vs. 59%, respectively, p<0.001).…”

“…This therapy abolishes the hepatic source of amyloidogenic TTR variants in serum and progression of the disease has at least halted in the patients receiving The prognosis of FAP patients after liver transplantation seems to be dependent on their preoperative state (19). FAPWTR emphasized that the severity of autonomic symptoms significantly affected the patient's recovery after operation, because many usually appeared in the patients at later stages of FAP (15). However, gastrointestinal autonomic symptoms, including severe episodic nausea, vomiting, and alternating constipation and diarrhea, are often initial symptoms in Japanese patients with this disease (1), and these bowel dysfunctions improved shortly after transplantation in the FAP patients in our series (11,18).…”

Transthyretin-Related Familial Amyloid Polyneuropathy: In the Light of New Developments

“…Known as a fatal disease, the first and an important development in the treatment of TTR-FAP was orthotopic liver transplantation, which was shown to have prognostic effects on TTR-FAP (22). Liver transplantation was performed for years in more than 2000 patients and as a result of removing the source of TTR, there was a 90% decrease in serum levels of TTR (23).…”