Recommendations for myasthenia gravis clinical trials

被引：129

作者：

Benatar, Michael ^{[2
]}

Sanders, Donald B. ^{[1
]}

Burns, Ted M. ^{[3
]}

Cutter, Gary R. ^{[4
]}

Guptill, Jeffrey T. ^{[1
]}

Baggi, Fulvio ^{[5
]}

Kaminski, Henry J. ^{[6
]}

Mantegazza, Renato

Meriggioli, Matthew N. ^{[7
]}

Quan, Joanne ^{[8
]}

Wolfe, Gil I. ^{[9
]}

机构：

[1] Duke Univ, Med Ctr, Div Neurol, DUMC 3403, Durham, NC 27710 USA

[2] Univ Miami, Sch Med, Dept Neurol, Miami, FL USA

[3] Univ Virginia, Sch Med, Dept Neurol, Charlottesville, VA 22908 USA

[4] Univ Alabama Birmingham, Sch Publ Hlth, Birmingham, AL 35294 USA

[5] Fdn Ist Neurol Carlo Besta, Milan, Italy

[6] George Washington Univ, Sch Med, Dept Neurol, Washington, DC USA

[7] Univ Illinois, Coll Med, Dept Neurol & Rehabil, Chicago, IL USA

[8] Bayhill Therapeut Inc, Palo Alto, CA USA

[9] SUNY Buffalo, Dept Neurol, SUNY Sch Med & Biomed Sci, Buffalo, NY 14260 USA

来源：

MUSCLE & NERVE | 2012年 / 45卷 / 06期

关键词：

all clinical trials; clinical trials methodology; study design; myasthenia; outcome measures; quality of life; QUALITY-OF-LIFE; INTRAVENOUS IMMUNOGLOBULIN; DOUBLE-BLIND; MYCOPHENOLATE-MOFETIL; PLASMA-EXCHANGE; AZATHIOPRINE; CYCLOSPORINE; INSTRUMENT; SCORE; DRUG;

D O I：

10.1002/mus.23330

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

100204 [神经病学];

摘要：

The recommendations for clinical research standards published in 2000 by a task force of the Medical Scientific Advisory Board (MSAB) of the Myasthenia Gravis Foundation of America (MGFA) were largely successful in introducing greater uniformity in the recording and reporting of MG clinical trials. Recognizing that changes in clinical trial design and implementation may increase the likelihood that new therapies are developed for MG, the MGFA MSAB Task Force here presents updated recommendations for the design and implementation of clinical trials in MG, including (a) the use of a quantitative measure, such as the MG-Composite, that is weighted for clinical significance and incorporates patient reported outcomes; (b) consideration of nontrial strategies; and (c) development of biomarkers that support mechanistic studies of pharmacotherapies. The hope is that these updated task force recommendations will expedite the development and acceptance of more effective and less noxious therapies for MG. Muscle Nerve 45: 909917, 2012

引用

页码：909 / 917

页数：9

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