title: The Diflunisal Trial: study accrual and drug tolerance.
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.
authors.affiliation: Department of Medicine & Data Coordinating Center, Boston University Medical Center, Boston, MA 02118, USA. jberk@bu.edu
authors.forename: John L
authors.initials: JL
authors.lastname: Berk
authors.forename: Ole B
authors.initials: OB
authors.lastname: Suhr
authors.forename: Yoshiki
authors.initials: Y
authors.lastname: Sekijima
authors.forename: Taro
authors.initials: T
authors.lastname: Yamashita
authors.forename: Michael
authors.initials: M
authors.lastname: Heneghan
authors.forename: Steven R
authors.initials: SR
authors.lastname: Zeldenrust
authors.forename: Yukio
authors.initials: Y
authors.lastname: Ando
authors.forename: Shu-ichi
authors.initials: S
authors.lastname: Ikeda
authors.forename: Peter
authors.initials: P
authors.lastname: Gorevic
authors.forename: Giampaolo
authors.initials: G
authors.lastname: Merlini
authors.forename: Jeffrey W
authors.initials: JW
authors.lastname: Kelly
authors.forename: Martha
authors.initials: M
authors.lastname: Skinner
authors.forename: Alice B
authors.initials: AB
authors.lastname: Bisbee
authors.forename: Peter J
authors.initials: PJ
authors.lastname: Dyck
authors.forename: Laura
authors.initials: L
authors.lastname: Obici
mesh_terms: D000328:Adult; D000368:Aged; D028227:Amyloid Neuropathies, Familial / Q000188:drug therapy*; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000009:adverse effects / Q000627:therapeutic use*; D004061:Diflunisal / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D055815:Young Adult
chemical_list: D000894:Anti-Inflammatory Agents, Non-Steroidal; D004061:Diflunisal