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Transplant of Partial Livers to Treat a Rare Nerve Disorder FREE

[+] Article and Author Information

The summary below is from the full report titled “Partial-Liver Transplantation To Treat Familial Amyloid Polyneuropathy: Follow-up of 11 Patients.” It is in the 19 October 1999 issue of Annals of Internal Medicine (volume 131, pages 592-595). The authors are Y. Takei, S. Ikeda, Y. Hashikura, T. Ikegami, and S. Kawasaki.


Ann Intern Med. 1999;131(8):592. doi:10.7326/0003-4819-131-8-199910190-00042
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What is the problem and what is known about it so far?

Familial amyloid polyneuropathy (FAP) is a rare disease where a substance called amyloid builds up in nerves and leads to nerve damage. This nerve damage can lead to muscular weakness and can interfere with the nerves that help internal organs work properly. The disease is known to be a result of an abnormal gene that controls the way the body handles a substance (transthyretin) that can lead to amyloid build up. The liver is the body organ that makes most of the transthyretin in the body. Experts on FAP believe that replacing the liver with all or part of a liver without this abnormal gene might be a way to treat FAP.

Why did the researchers do this particular study?

The researchers describe what happened to a series of patients with FAP who had their livers replaced with parts of normal livers (partial liver transplantation).

Who was studied?

Eleven patients with FAP who were receiving care at a special center in Japan and had partial liver transplants.

How was the study done?

Between 1993 and 1998, these 11 patients had partial liver transplantations. The parts of the normal livers were donated by living spouses or siblings of the patients. Before surgery all of the patients underwent physical examination, blood tests, and tests to measure nerve function in their arms and legs. They also had small specimens (biopsies) taken of their nerves so that the researchers could look for changes in the nerve cells.

What did the researchers find?

Of the 11 patients, 7 had nerve damage only in their digestive tract or legs for less than 4 years and all seven improved after the transplant. The remaining four patients had involvement in both their arms and legs and had been sick for more than 6 years. Three of these four patients did not do well after transplant; two of them died.

What were the limitations of the study?

The researchers studied only 11 patients and did not compare how these patients did compared to patients who did not have liver transplants. Many patients with FAP are not candidates for liver transplant.

What are the implications of the study?

Partial liver transplant may be a reasonable treatment for FAP. It seems that patients who have less neurologic damage and who have been sick with FAP for a shorter period of time do the best after this procedure.

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