Journal
NEUROMUSCULAR DISORDERS
Volume 25, Issue 12, Pages 937-944Publisher
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.nmd.2015.09.012
Keywords
Rating scale; Patient-reported outcome; Functional ability; Rasch analysis; Costs; Utilities
Categories
Funding
- GlaxoSmithKline [HO-11-794]
- European Commission [036825]
- Second Public Health Programme [2012 3307]
- AFM [16104]
- German Duchenne Parent Project aktion benni co
- Deutsche Gesellschaft fur Muskelkranke (DGM) e.V.
- Action Duchenne, a patient led charity
- Parent Project Muscular Dystrophy (PPMD)
- Medical Research Council as part of the MRC Centre for Neuromuscular Diseases [G1002274, 98482]
- MRC [MR/K000608/1, G1002274] Funding Source: UKRI
- Medical Research Council [MR/K000608/1, G1002274] Funding Source: researchfish
- National Institute for Health Research [NF-SI-0512-10036] Funding Source: researchfish
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The objective of this study was to describe the development and initial psychometric analysis of the UK English version of the Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT), a patient-reported outcome (PRO) scale designed to measure functional ability in patients with Duchenne muscular dystrophy (DMD). Item selection was made by neuromuscular specialists and a Rasch analysis was performed to understand the psychometric properties of the DMDSAT. Instrument scores were also linked to cost of illness and health-related quality of life data. The administered version, completed by 186 UK patient-caregivers pairs, included eight items in four domains: Arm function, Mobility, Transfers, and Ventilation status. These items together successfully operationalized functional ability in DMD, with excellent targeting and reliability (Person Separation Index: 0.95; Cronbach's alpha: 0.93), stable item locations, and good fit to the Rasch model (mean person/item fit residual: -0.211/-0.44, SD: 0.32/1.28). Estimated item difficulty was in excellent agreement with clinical opinion (Spearman's rho: 0.95) and instrument scores mapped well onto health economic outcomes. We show that the DMDSAT is a PRO instrument fit for purpose to measure functional ability in ambulant and non-ambulant patients with DMD. Rasch analysis augments clinical expertise in the development of robust rating scales. (C) 2015 The Authors. Published by Elsevier B.V.
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