Partial-Liver Transplantation To Treat Familial Amyloid Polyneuropathy: Follow-up of 11 Patients

19 October 1999 | Volume 131 Issue 8 | Pages 592-595

Background: Recently, liver transplantation has been used to treat patients with familial amyloid polyneuropathy (FAP).

Objective: To describe the clinical course of patients with FAP who received partial-liver transplantation from living donors.

Design: Case series.

Setting: University hospital in Matsumoto, Japan.

Patients: 11 patients with FAP who underwent partial-liver transplantation. The transthyretin gene abnormality in all 11 patients was the substitution of methionine for valine at position 30.

Intervention: Partial liver transplantation from living donors.

Measurements: Preoperative and follow-up (3 to 64 months) clinical data, including routine laboratory data, nerve conduction velocity tests, and sural nerve histology.

Results: All 7 patients who had severe gastrointestinal autonomic disorders or polyneuropathy localized to the lower limbs for less than 4 years showed improvement. Three of 4 patients with polyneuropathy involving both the upper and lower limbs had adverse outcomes, including two deaths. The preoperative duration of their illness was more than 6 years. These 3 patients also had marked decreases in creatinine clearance and nerve conduction velocities and severe loss of myelinated fibers in sural nerves.

Conclusion: Preoperative clinical severity and duration of illness are associated with outcomes after liver transplantation for FAP.

Author and Article Information

From Shinshu University School of Medicine, Matsumoto, Japan.

Grant Support: In part by a research grant from the Intractable Disease Division, Ministry of Health and Welfare, Primary Amyloidosis Research Committee, Japan; a group research grant for the pathogenesis and therapy for hereditary neuropathy (10B-3); and a Grant-in-Aid for Scientific Research, Ministry of Education, Science and Culture, Japan (07457124).

Requests for Reprints: Shu-ichi Ikeda, MD, Department of Medicine, Neurology, Shinshu University School of Medicine, Matsumoto 390-8621, Japan; e-mail, ikedasi{at}hsp.md.shinshu-u.ac.jp.

Current Author Addresses: Drs. Takei and Ikeda: Department of Medicine, Neurology, Shinshu University School of Medicine, Matsumoto 390-8621, Japan.

Drs. Hashikura, Ikegami, and Kawasaki: Department of Surgery, Shinshu University School of Medicine, Matsumoto 390-8621, Japan.