The Diflunisal Trial: Study accrual and drug tolerance

被引：36

作者：

Berk, John L. ^{[1
,2
]}

Suhr, Ole B. ^{[3
]}

Sekijima, Yoshiki ^{[4
]}

Yamashita, Taro ^{[5
]}

Heneghan, Michael ^{[6
]}

Zeldenrust, Steven R. ^{[7
,8
]}

Ando, Yukio ^{[5
]}

Ikeda, Shu-ichi ^{[4
]}

Gorevic, Peter ^{[9
]}

Merlini, Giampaolo ^{[10
]}

Kelly, Jeffrey W. ^{[11
]}

Skinner, Martha ^{[2
]}

Bisbee, Alice B. ^{[2
]}

Dyck, Peter J. ^{[7
,8
]}

Obici, Laura ^{[11
]}

机构：

[1] Boston Univ, Med Ctr, Amyloid Treatment & Res Program, Dept Med, Boston, MA 02118 USA

[2] Boston Univ, Med Ctr, Data Coordinating Ctr, Boston, MA 02118 USA

[3] Umea Univ, Dept Med, Umea, Sweden

[4] Shinshu Univ, Sch Med, Dept Med 3, Matsumoto, Nagano 390, Japan

[5] Kumamoto Univ, Grad Sch Med Sci, Kumamoto, Japan

[6] Kings Coll Hosp London, Liver Unit, London, England

[7] Mayo Clin, Dept Neurol, Rochester, MN USA

[8] Mayo Clin, Dept Med, Rochester, MN USA

[9] Mt Sinai Med Ctr, Dept Med, New York, NY 10029 USA

[10] Univ Pavia, IRCCS Policlin San Matteo, I-27100 Pavia, Italy

[11] Scripps Res Inst, La Jolla, CA 92037 USA

来源：

AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS | 2012年 / 19卷

关键词：

Clinical trials; diflunisal; FAP; neuropathy; transthyretin; LIVER-TRANSPLANTATION; POLYNEUROPATHY; TRANSTHYRETIN;

D O I：

10.3109/13506129.2012.678509

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

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.

引用

页码：37 / 38

页数：2

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