Dataset Information

Disability Outcome Measures in Phase III Clinical Trials in Multiple Sclerosis.

ABSTRACT: Accumulating neurological disability has a substantial impact on the lives of patients with multiple sclerosis (MS). As well as the established Expanded Disability Status Scale (EDSS), several other outcome measures are now available for assessing disability progression in MS. This review extends the findings of a previous analysis of relapsing-remitting MS (RRMS) trials published up to 2012, to determine whether there has been a shift in outcome measures used to assess disability in phase III clinical trials in RRMS and progressive MS. Forty relevant trials were identified (RRMS, n = 16; progressive MS, n = 18; other/mixed phenotypes, n = 6). Sustained EDSS worsening, particularly over 3 months, was included as an endpoint in almost all identified trials. Other disability-related endpoints included the Multiple Sclerosis Functional Composite z-score and scores for the physical component summary of the Multiple Sclerosis Impact Scale and Medical Outcomes Study Short-Form (36-item) Health Survey. Tests assessing manual dexterity, ambulation, vision and cognition were also employed, and in some trials, composite endpoints were used. However, there was no obvious trend in choice of disability outcome measures over time. Sustained EDSS worsening over short time periods continues to be the most widely used measure of disability progression in pivotal MS trials, despite its well-recognised limitations. A new tool set is needed for use in MS clinical trials that detects the benefit of potential treatments that slow (or reverse) progressive disability.

SUBMITTER: Uitdehaag BMJ

PROVIDER: S-EPMC6061412 | biostudies-literature | 2018 Jun

REPOSITORIES: biostudies-literature

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Disability Outcome Measures in Phase III Clinical Trials in Multiple Sclerosis.

Uitdehaag Bernard M J BMJ

CNS drugs 20180601 6

Accumulating neurological disability has a substantial impact on the lives of patients with multiple sclerosis (MS). As well as the established Expanded Disability Status Scale (EDSS), several other outcome measures are now available for assessing disability progression in MS. This review extends the findings of a previous analysis of relapsing-remitting MS (RRMS) trials published up to 2012, to determine whether there has been a shift in outcome measures used to assess disability in phase III c ...[more]

PMID: 29926371

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Project description:BackgroundMultiple Sclerosis (MS) is associated with impaired gait and a growing number of clinical trials have investigated efficacy of various interventions. Choice of outcome measures is crucial in determining efficiency of interventions. However, it remains unclear whether there is consensus on which outcome measures to use in gait intervention studies in MS.ObjectiveWe aimed to identify the commonly selected outcome measures in randomized controlled trials (RCTs) on gait rehabilitation interventions in people with MS. Additional aims were to identify which of the domains of the International Classification of Functioning, Disability and Health (ICF) are the most studied and to characterize how outcome measures are combined and adapted to MS severity.MethodsPubmed, Cochrane Central, Embase and Scopus databases were searched for RCT studies on gait interventions in people living with MS according to PRISMA guidelines.ResultsIn 46 RCTs, we identified 69 different outcome measures. The most used outcome measures were 6-minute walking test and the Timed Up and Go test, used in 37% of the analyzed studies. They were followed by gait spatiotemporal parameters (35%) most often used to inform on gait speed, cadence, and step length. Fatigue was measured in 39% of studies. Participation was assessed in 50% of studies, albeit with a wide variety of scales. Only 39% of studies included measures covering all ICF levels, and Participation measures were rarely combined with gait spatiotemporal parameters (only two studies).ConclusionsSelection of outcome measures remains heterogenous in RCTs on gait rehabilitation interventions in MS. However, there is a growing consensus on the need for quantitative gait spatiotemporal parameter measures combined with clinical assessments of gait, balance, and mobility in RCTs on gait interventions in MS. Future RCTs should incorporate measures of fatigue and measures from Participation domain of ICF to provide comprehensive evaluation of trial efficacy across all levels of functioning.

Dataset Information

Disability Outcome Measures in Phase III Clinical Trials in Multiple Sclerosis.

Publications

Disability Outcome Measures in Phase III Clinical Trials in Multiple Sclerosis.

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