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Efficacy of diflunisal on autonomic dysfunction of late-onset familial amyloid polyneuropathy (TTR Val30Met) in a Japanese endemic area.

AbstractOBJECTIVE:
To evaluate the long-term efficacy and safety of diflunisal in late-onset familial amyloid polyneuropathy (FAP) in a Japanese endemic area.
METHODS:
Consecutive six FAP patients (mean age: 65.8 ± 7.3 years) with a transthyretin (TTR) Val30Met mutation from an endemic area of late-onset FAP were prospectively recruited to an open label study with oral diflunisal (250 mg twice a day). We evaluated clinical symptoms, Kumamoto FAP score, modified body mass index (mBMI), Medical Research Council sum score, nerve conduction studies (NCS), electrocardiogram (ECG), ECG Holter monitor test, echocardiography, and (123)iodine-metaiodobenzylguanidine ((123)I-MIBG) myocardial scintigraphy.
RESULTS:
One patient ceased to take diflunisal because of hematuria which was reversible. The other five patients were treated with diflunisal for 3-5 (4.4 ± 0.9 years) years. Autonomic symptoms (orthostatic hypotension and gastrointestinal symptoms) disappeared after treatment in two of the four patients with the symptoms. Delayed heart to mediastinum ratio on (123)I-MIBG imaging, a marker of cardiac postganglionic sympathetic nerve function, increased during the three-year treatment. mBMI was maintained through observation period. While, motor and sensory symptoms, Kumamoto FAP scores, and data on NCS gradually deteriorated.
CONCLUSION:
Diflunisal might be effective especially for autonomic dysfunction in late-onset FAP with a TTR Val30Met mutation.
AuthorsRyoichi Takahashi, Kenjiro Ono, Shutaro Shibata, Keiko Nakamura, Junji Komatsu, Yoshihisa Ikeda, Tokuhei Ikeda, Miharu Samuraki, Kenji Sakai, Kazuo Iwasa, Daiki Kayano, Masahito Yamada
JournalJournal of the neurological sciences (J Neurol Sci) Vol. 345 Issue 1-2 Pg. 231-5 (Oct 15 2014) ISSN: 1878-5883 [Electronic] Netherlands
PMID25060417 (Publication Type: Journal Article)
CopyrightCopyright © 2014 Elsevier B.V. All rights reserved.
Chemical References
  • Anti-Inflammatory Agents, Non-Steroidal
  • Prealbumin
  • Radiopharmaceuticals
  • 3-Iodobenzylguanidine
  • Diflunisal
  • Methionine
  • Valine
Topics
  • 3-Iodobenzylguanidine
  • Aged
  • Amyloid Neuropathies, Familial (complications, genetics)
  • Anti-Inflammatory Agents, Non-Steroidal (therapeutic use)
  • Autonomic Nervous System Diseases (diagnostic imaging, drug therapy, etiology)
  • Diflunisal (therapeutic use)
  • Electrocardiography
  • Female
  • Humans
  • Japan
  • Male
  • Methionine (genetics)
  • Middle Aged
  • Mutation (genetics)
  • Pharmacogenetics
  • Prealbumin (genetics)
  • Radionuclide Imaging
  • Radiopharmaceuticals
  • Valine (genetics)

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