RASCH ANALYSIS OF CLINICAL OUTCOME MEASURES IN SPINAL MUSCULAR ATROPHY

被引：57

作者：

Cano, Stefan J. ^{[1
]}

Mayhew, Anna ^{[2
]}

Glanzman, Allan M. ^{[3
]}

Krosschell, Kristin J. ^{[4
]}

Swoboda, Kathryn J. ^{[5
]}

Main, Marion ^{[6
]}

Steffensen, Birgit F. ^{[7
]}

Berard, Carole ^{[8
]}

Girardot, Francoise ^{[9
]}

Payan, Christine A. M. ^{[9
]}

Mercuri, Eugenio ^{[10
]}

Mazzone, Elena ^{[10
]}

Elsheikh, Bakri ^{[11
]}

Florence, Julaine ^{[12
]}

Hynan, Linda S. ^{[13
,14
]}

Iannaccone, Susan T. ^{[15
]}

Nelson, Leslie L. ^{[16
]}

Pandya, Shree ^{[17
]}

Rose, Michael ^{[18
]}

Scott, Charles ^{[19
]}

Sadjadi, Reza ^{[20
]}

Yore, Mackensie A. ^{[21
]}

Joyce, Cynthia ^{[21
]}

Kissel, John T. ^{[22
,23
]}

机构：

[1] Peninsula Coll Med & Dent, Clin Neurol Res Grp, Plymouth, Devon, England

[2] Int Ctr Life, Newcastle Muscle Team, Inst Human Genet, Newcastle, NSW, Australia

[3] Childrens Hosp Philadelphia, Dept Phys Therapy, Philadelphia, PA 19104 USA

[4] Northwestern Univ, Feinberg Sch Med, Dept Phys Therapy & Human Movement Sci, Chicago, IL 60611 USA

[5] Univ Utah, Sch Med, Salt Lake City, UT USA

[6] Great Ormond St Childrens Hosp, Dubowitz Neuromuscular Ctr, London, England

[7] Rehabiliterings Ctr Muskelsvind, Aarhus, Denmark

[8] Ctr Hosp, Lyon, France

[9] Hop La Pitie Salpetriere, AP HP, Dept Clin Pharmacol, Paris, France

[10] Polclin Gemell, Dept Paediat Neurol, Rome, Italy

[11] Ohio State Univ, Dept Neurol, Div Neuromuscular Med, Columbus, OH 43210 USA

[12] Washington Univ, Sch Med, Dept Neurol, Neuromuscular Div, St Louis, MO 63110 USA

[13] Univ Texas SW Med Ctr Dallas, Dept Clin Sci Biostat, Dallas, TX 75390 USA

[14] Univ Texas SW Med Ctr Dallas, Dept Psychiat, Dallas, TX 75390 USA

[15] Univ Texas SW Med Ctr Dallas, Childrens Med Ctr Ambulatory Care Pavil, Dallas, TX 75390 USA

[16] Univ Texas SW Med Ctr Dallas, Dept Phys Therapy, Dallas, TX 75390 USA

[17] Univ Rochester, Sch Med & Dent, Dept Neurol, Rochester, NY 14642 USA

[18] Kings Coll Hosp London, Dept Neurol, London, England

[19] CBS Squared Inc, PA Project Cure, Ft Washington, MD USA

[20] Univ Virginia, Dept Neurol, Charlottesville, VA USA

[21] Spinal Muscular Atrophy Fdn, New York, NY USA

[22] Ohio State Univ, Dept Neurol, Nationwide Childrens Hosp, Columbus, OH 43210 USA

[23] Ohio State Univ, Dept Pediat, Nationwide Childrens Hosp, Columbus, OH 43210 USA

来源：

MUSCLE & NERVE | 2014年 / 49卷 / 03期

关键词：

clinical trials; motor neuron disease; outcome measures; Rasch analysis; spinal muscular atrophy; FUNCTIONAL MOTOR SCALE; RATING-SCALES; RELIABILITY; VALIDATION; DYSTONIA; CHILDREN; MODELS;

D O I：

10.1002/mus.23937

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Introduction: Trial design for SMA depends on meaningful rating scales to assess outcomes. In this study Rasch methodology was applied to 9 motor scales in spinal muscular atrophy (SMA). Methods: Data from all 3 SMA types were provided by research groups for 9 commonly used scales. Rasch methodology assessed the ordering of response option thresholds, tests of fit, spread of item locations, residual correlations, and person separation index. Results: Each scale had good reliability. However, several issues impacting scale validity were identified, including the extent that items defined clinically meaningful constructs and how well each scale measured performance across the SMA spectrum. Conclusions: The sensitivity and potential utility of each SMA scale as outcome measures for trials could be improved by establishing clear definitions of what is measured, reconsidering items that misfit and items whose response categories have reversed thresholds, and adding new items at the extremes of scale ranges. Muscle Nerve49:422-430, 2014

引用

页码：422 / 430

页数：9

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