LIVER TRANSPLANTATION FOR FAMILIAL AMYLOIDOTIC POLYN EURO PATH Y DOES NOT PREVENT DISEASE PROGRESSION IN A MAJORITY OF PATIENTS

摘要

Aim: To determine the outcome in patients transplanted for FAP.Methods: We reviewed the medical records of all patients undergoing OLT for FAP at our institution between 11/96 and 12/02. Information collected included: sex, mutation, age, clinical manifestations, and patient and aiiograft survival.Results: We evaluated 12 males with a mean age of 58 years (range 47-68). Six received liver, and six patients liver and heart transplants. TTR mutations included MET-30 (n=4), ALA-60 (n=4), TYR-77 (N=2), GLY-42 (n=1) and LYS-89 (n=1). Mean time from symptoms to diagnosis was 3.4 years (0-10). Patients were listed for OLT an average of 10.8 months (0-60) after diagnosis. Mean time from listing to OLT was 8.2 months (1-18). Five patients died after OLT, during a mean follow-up of 3.5 years (1-6). Survival was 100% (1 year) and 92% (3 year). Symptoms worsened after OLT in seven, and improved or stabilized in five. Following OLT, neuropathy improved in four patients, worsened in seven and was unchanged in one. Prior to OLT, all twelve patients had an increased IVST on echocardiogram. Post-OLT cardiac symptoms improved in six (five had cardiac transplantation), worsened in three, and were unchanged in three (one had cardiac transplantation). Post-OLT echocardiograms were available in 4 of 6 patients who had not received a cardiac transplant. Echocardiograms were performed an average of 29.3 months (12-36) post-transplant and IVST improved in one, worsened in two, and remained the same in one. Conclusions: The benefits of OLT in FAP, particularly in non-MET-30 patients, remain indeterminate.