Outcome measures in immune‐mediated neuropathies: the need to standardize their use and to understand the clinimetric essentials

Journal of the Peripheral Nervous System - Tập 13 Số 2 - Trang 136-147 - 2008
Sonja I. van Nes1,2,3, Catharina G. Faber1,2,3, Ingemar S.J. Merkies1,2,3
11 Department of Neurology, Erasmus Medical Centre, Rotterdam
22Department of Neurology, Maastricht University Medical Centre, Maastricht
33Department of Neurology, Spaarne Hospital, Hoofddorp, The Netherlands

Tóm tắt

Abstract  Peripheral neurological disorders like neuropathies may cause impairments (such as weakness and sensory deficits), which may lead to problems in daily life and social functioning with a possible decrement in quality of life expectations. Choosing the proper outcome measure to evaluate the therapeutic efficacy of an intervention at one of these levels of outcome should therefore be considered as fundamental to the design of randomized trials in peripheral neurological disorders. However, these choices are dependent not only on the proposed research purposes but also, and perhaps more importantly, on the fulfillment of the scientific needs of these measures. With an increasing demand for accuracy, a thorough and comprehensive evaluation of an outcome measure is needed to determine its simplicity, communicability, validity, reliability, and responsiveness before being clinically applicable, techniques that are being captured by the science of clinimetrics. Most neurologists are still unfamiliar with these rigorous methodological essentials or overlook some of them in their trial preparations because these are considered time consuming and mind numbing. This review will highlight, against the background of the international classification framework and clinimetric needs for outcome measures, the selected scales applied in published randomized controlled trials in patients with Guillain‐Barré syndrome, chronic inflammatory demyelinating polyradiculoneuropathy, multifocal motor neuropathy, and gammopathy‐related neuropathies. The need for comparison responsiveness studies between equally valid and reliable measures and to standardize their use is emphasized in these conditions. Finally, specific recommendations are given to move from classic to modern clinimetric approach when constructing, evaluating, and selecting outcome measures using new methods like Rasch analysis, accentuating the need of shifting toward a more modern era.

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Tài liệu tham khảo

Aaronson NK, 1988, Quality of life: what is it? How should it be measured?, Oncology, 2, 69

10.1097/01.mlr.0000103528.48582.7c

10.1053/apmr.2003.50247

Bond TG, 2001, Applying the Rasch Model: Fundamental Measurement for the Human Sciences, 29, 10.4324/9781410600127

10.1212/WNL.46.1.100

10.1007/s00415-002-0808-z

10.1002/ana.410400516

10.1212/WNL.35.8.1173

10.1056/NEJM199111213252105

10.1212/WNL.41.6.799

10.1212/01.WNL.0000149522.32823.EA

10.1016/0168-8510(90)90421-9

10.1212/WNL.37.5.837

10.1212/WNL.55.9.1256

Feinstein AR, 1987, Clinimetrics, 1, 10.2307/j.ctt1xp3vbc

10.3310/hta2140

10.1002/ana.410220612

10.1002/ana.410410304

Garssen MP, 2007, Treatment of Guillain‐Barré syndrome with mycophenolate mofetil: a pilot study, J Neurol Neurosurg Psychiatry, 78, 1416

10.1136/jnnp.2004.046227

10.1136/jnnp.2005.081547

10.1016/S0140-6736(84)91341-2

Guillain‐Barré Syndrome Steroid Trial Group, 1993, Double‐blind trial of intravenous methylprednisolone in Guillain‐Barré syndrome, Lancet, 341, 586

10.1212/WNL.35.8.1096

10.3109/00365549509019016

10.1212/WNL.53.1.57

10.1093/brain/119.4.1067

10.1093/brain/119.4.1055

10.1016/0022-510X(95)00357-8

10.1097/00005650-200009002-00007

10.1136/jnnp.60.2.127

10.1111/j.1749-6632.1998.tb11015.x

10.1002/ana.1088

10.1016/S0140-6736(78)92644-2

10.1016/S1474-4422(07)70329-0

10.1097/00005650-198903001-00015

10.1002/mus.880141111

10.1016/S0140-6736(03)15324-X

10.1542/peds.2004-1324

10.1093/brain/124.1.145

Liang MH, 1995, Evaluating measurement responsiveness, J Rheumatol, 22, 1191

10.1097/00005650-199007000-00008

10.1212/01.WNL.0000044402.16315.FC

10.1016/j.nmd.2007.08.004

10.1136/jnnp.63.1.28

10.1097/00006324-200208000-00015

10.1056/NEJM199204233261705

Medical Research Council, 1943, Her Majesty’s Stationery Office, 1

10.1212/WNL.35.11.1551

10.1212/WNL.56.4.445

10.1212/WNL.53.8.1648

10.1002/1097-4598(200009)23:9<1393::AID-MUS10>3.0.CO;2-O

10.1212/WNL.59.1.84

MerkiesISJ(2001).Evaluation of scales and measurement instruments in immune‐mediated polyneuropathies[thesis].Erasmus Medical Centre Rotterdam The Netherlands. ISBN: 90‐9014393‐9.

10.1016/j.nmd.2005.12.003

10.1212/WNL.54.4.943

10.1136/jnnp.72.5.596

10.1212/01.wnl.0000265055.28218.cc

Nomura T, 2001, A randomized controlled trial comparing intravenous immunoglobulin and plasmapheresis in Guillain‐Barré syndrome, Neurol Ther, 18, 69

Nunnally JC, 1978, Psychometric Theory, 244

10.1136/jnnp.59.3.243

10.1016/S0140-6736(84)90819-5

10.1097/00000539-200212000-00046

10.1093/brain/awm144

10.1016/S0140-6736(96)09095-2

10.1212/01.WNL.0000092019.53628.88

10.1136/jnnp.71.2.235

Shukla SK, 1988, Double blind control trial of prednisolone in Guillain‐Barré syndrome – a clinical study, Clin India, 52, 128

Singh NK, 1996, Do corticosteroids influence the disease course or mortality in Guillain‐Barré syndrome, J Assoc Physicians India, 44, 22

Streiner DL, 1998, A Practical Guide to Their Development and Use

10.1212/WNL.26.3.205

10.1136/jnnp.59.3.248

10.1212/WNL.40.2.209

10.1136/jnnp.56.1.36

Wang R, 2001, Intravenous immunoglobulin in children with Guillain‐Barré syndrome, J Appl Clin Pediatr, 16, 223

Ware JE, 1997, SF‐36 Health Survey Manual and Interpretation Guide

10.1212/WNL.57.5.774

World Health Organization, 1980, International Classification of Impairments, Disabilities, and Handicaps, 7

World Health Organization, 2001, International Classification of Functioning, Disability, and Health, 3

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Thông tin xuất bản

Journal of the Peripheral Nervous System

Tập 13 Số 2

136-147

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