[Autonomic dysfunction in FAP: its therapeutic effect by liver transplantation].
FAP is characterized by progressive polyneuropathy and autonomic dysfunction and the latter consists of marked orthostatic hypotension, disturbed bowel movement, impotence and urinary incontinence. All these autonomic symptoms severely affect patient's daily activity. The precursor protein of amyloid fibrils in this disease is a variant form of transthyretin (TTR) in serum. Since TTR is produced mainly in the liver, liver transplantation has been employed for FAP patients as only one curative treatment. During the past 12 years more than 50 FAP patients underwent liver transplantation in Japan and the five-year survival rate of them was 77%. Early intervention (less than 5 years after onset) can provide a better chance of improving patients' condition after transplantation and gastrointestinal autonomic symptoms that include severe episodic nausea, vomiting, and alternating constipation and diarrhea significantly relieve shortly after operation. Preoperative clinical severity and the nutritional status of patients are correlated with their outcome after liver transplantation. Among them the presence of an autonomic failure in FAP patients seems to be contraindication for this challenging operation.
Ikeda
Shu-ichi
S
Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine.
D000328:Adult; D028227:Amyloid Neuropathies, Familial / Q000628:therapy*; D001342:Autonomic Nervous System Diseases / Q000150:complications*; D005260:Female; D006801:Humans; D016031:Liver Transplantation; D008297:Male