The Diflunisal Trial: study accrual and drug tolerance.

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.

Citation

John L Berk, Ole B Suhr, Yoshiki Sekijima, Taro Yamashita, Michael Heneghan, Steven R Zeldenrust, Yukio Ando, Shu-ichi Ikeda, Peter Gorevic, Giampaolo Merlini, Jeffrey W Kelly, Martha Skinner, Alice B Bisbee, Peter J Dyck, Laura Obici, Familial Amyloidosis Consortium. The Diflunisal Trial: study accrual and drug tolerance. Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis. 2012 Jun;19 Suppl 1:37-8

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PMID: 22551208

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