Liver transplants can significantly prolong survival in familial amyloid polyneuropathy (FAP) patients, and the longer they must wait for the transplant may mean the disease will worsen after the procedure.
Those findings come from the study “Liver Transplantation for Familial Amyloid Polyneuropathy (Val30Met): Long-Term Follow-up Prospective Study in a Nontransplant Center,” which was published in Transplantation Proceedings.
Because the liver produces most of the mutated transthyretin that causes FAP, liver transplant has been considered the standard FAP treatment since the 1990s. It has been observed that disease duration has an important impact on disease progression, and that survival decreases with longer waiting periods before transplant.
In this study researchers in Greece reported long-term results for 12 FAP patients who received liver transplants and followed up on at their clinic.
The patients were an equal number of males and females and had an average age of 43 years. They were followed for 100 months (just over eight years), monitored continuously, and treated by a multi-disciplinarian team of doctors. All the patients also received drugs to suppress the immune system, which is common in transplants because it decreases the likelihood the body will reject the organ.
In their study, researchers detailed the clinical features of this group of patients. All of them had peripheral neuropathy (damage in the peripheral nerves), with 25% having severe damage to these nerves.
Nine patients (75%) had gastrointestinal problems such as constipation and diarrhea. About a third of the patients had eye problems like glaucoma, and two required foot amputations due to ulcers. The investigators stressed that clinicians need to pay attention to the feet of FAP patients, as they do for diabetes patients. “We emphasize the importance of the amyloid foot, which should receive special attention and be managed as the diabetic foot,” they stated.
Four of the patients waited more than a year after FAP diagnosis to receive a liver transplant, and the researchers noted these patients’ disease progression was particularly severe, with impaired nutrition and genital and urinary problems.
“Importantly,” they wrote in the paper, “the delay from diagnosis of FAP to liver transplant is associated with worse clinical outcomes.”
Over the course of the follow-up period, three of the patients died, though only one death was due to FAP (the other two were due to kidney disease and a traffic accident). Thus, the investigators concluded, “FAP patients have a long survival after liver transplantation.”
The investigators concluded their paper by stressing the importance of having a diverse medical team to treat this disease. “Since the disease is systematic and there are several organ system complications, these need to be treated by specialists,” they wrote.
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