Effect of Orthotopic Liver Transplantation on the Progression of Familial Amyloidotic Polyneuropathy

Abstract

Liver transplantation offers the only cure for the genetic defect causing FAP and appears to result in subjective and objective improvement in neurological dysfunction. Patients with preexisting cardiovascular abnormalities progress despite transplantation; therefore, consideration for combined heart-liver transplantation may be warranted in this subset of patients.

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

“…Recently, it was noted that after transplantation rapid deterioration of cardiac function with further thickening of the ventricular wall occurred in some FAP patients, although polyneuropathy and autonomic dysfunction were stabilized or slightly improved. These findings were originally obtained in non-Val30Met TTR type patients who may have had substantial amyloid deposition on the myocardium prior to the operation (24)(25)(26). However, a similar finding was reported in typical FAP patients with Val30Met TTR type (27).…”

Liver transplantation for hereditary transthyretin amyloidosis

“…37,56,79,107,110 However, onset of more pronounced nausea and vomiting has been noted in several of our patients after transplantation. Malabsorption of bile acids and fat and occurrence of bacterial overgrowth of the small bowel appear to be unchanged after transplantation.…”

“…78,79 For patients with a history of dizziness, syncope, or Holter ECG changes of sinoatrial or AV heart block, as well as for patients with autonomic circulatory disturbances, as mentioned previously, a pacemaker is indicated during transplantation; for other patients, a readiness to quickly insert a pacemaker during transplantation is sufficient.…”

“…Good survival and symptomatic response were noted in a German patient with the ATTR Lys47Gly mutation, 90 and good results with regard to both survival and symptomatic improvement were reported from Spain in a series of 4 patients with the Val71Ala mutation. 91 However, the mortality rate has been high for other mutations, and progression of cardiac amyloidosis after liver transplantation has been reported 31,32,79,92 (Table 1). A combined liver and heart transplantation may be needed in several mutations, although progression of both cardiac amyloidosis and polyneuropathy were reported for the ATTR Ser77Tyr mutation from 1 center.…”

“…31,78 However, for several non-ATTR Val30Met mutations, increased amyloid deposits have been noted after transplantation 31,78,79,92 (Table 1), and several patients have developed congestive heart failure. Survival has been poor for those patients, although cardiac failure has not been the most common cause of death.…”