シンポジウム１２‐２　末梢神経疾患研究の現在　家族性アミロイドポリニューロパチー（ＦＡＰ）の治療戦略

DOI Web Site PubMed 被引用文献1件 参考文献26件
  • 池田 修一
    信州大学医学部脳神経内科，リウマチ・膠原病内科

書誌事項

タイトル別名
  • Therapeutic strategy for familial amyloid polyneuropathy (FAP)
  • 家族性アミロイドポリニューロパチー(FAP)の治療戦略
  • カゾクセイ アミロイドポリニューロパチー FAP ノ チリョウ センリャク

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抄録

Familial amyloid polyneuropathy (FAP) was long considered to be an incurable disease, but a new therapeutic approach was developed 15 years ago. As the liver produces most of the transthyretin (TTR) in serum, it was assumed that the replacement of a liver expressing an abnormal TTR gene should stop the production of the variant TTR, the serum amyloid precursor in FAP. Until now about 1,500 FAP patients underwent liver transplantation, and the 10-year-survival rate is about 77%. After operation the progression of FAP symptoms certainly stopped, and patients who were in an early stage of the disease and underwent successful operations showed considerable improvement in their quality of life. Electrophysiological study of peripheral nerve function has demonstrated that liver transplantation can halt the progression of peripheral neuropathy in FAP patients, and histopathological regression of amyloid deposits was seen on the patients with long post-transplatation courses. Pharmacological therapies have been considered for FAP patients and among them, diflunisal, one of non-steroidal anti-inflammatory drugs, is very promising. TTR tetramer dissociation is an initial step for the process of TTR-derived amyloid fibril formation associated with FAP and diflinisal can inhibit this process by stabilization of the TTR tetramer. Clinical trial of this drug for FAP patients is now going worldwide.<br>

収録刊行物

  • 臨床神経学

    臨床神経学 49 (11), 953-955, 2009

    日本神経学会

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