The Diflunisal Trial: study accrual and drug tolerance

Amyloid. 2012 Jun:19 Suppl 1:37-8. doi: 10.3109/13506129.2012.678509. Epub 2012 May 2.

Abstract

Familial amyloidotic polyneuropathy (FAP) is a protein folding disorder that induces neuropathy and cardiomyopathy, leading to death within 7-15 years after onset of clinical disease. In vitro, small ligands binding the thyroid hormone docking site stabilize tetrameric transthyretin, inhibiting amyloid fibril formation. We undertook a randomized, placebo-controlled clinical trial to determine whether diflunisal, a well-known non-steroidal anti-inflammatory drug (NSAID) alters neurologic disease progression in FAP. We enrolled 130 subjects with wide age and FAP mutation representation. To date, few recognized complications of NSAIDs have occurred in the study cohort. Data collection will be completed by November 2012.

Publication types

  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Aged
  • Amyloid Neuropathies, Familial / drug therapy*
  • Anti-Inflammatory Agents, Non-Steroidal / adverse effects
  • Anti-Inflammatory Agents, Non-Steroidal / therapeutic use*
  • Diflunisal / adverse effects
  • Diflunisal / therapeutic use*
  • Female
  • Humans
  • Male
  • Middle Aged
  • Young Adult

Substances

  • Anti-Inflammatory Agents, Non-Steroidal
  • Diflunisal