RT Journal Article
SR Electronic
T1 Defining disability: development and validation of a mobility-Disability Severity Index (mDSI) in Charcot-Marie-tooth disease
JF Journal of Neurology, Neurosurgery & Psychiatry
JO J Neurol Neurosurg Psychiatry
FD BMJ Publishing Group Ltd
SP 635
OP 639
DO 10.1136/jnnp-2013-307390
VO 86
IS 6
A1 Sindhu Ramchandren
A1 Michael Shy
A1 Eva Feldman
A1 Ruth Carlos
A1 Carly Siskind
YR 2015
UL http://jnnp.bmj.com/content/86/6/635.abstract
AB Objective To develop and validate a reliable patient-reported scale that grades the severity of disability in Charcot-Marie-tooth disease (CMT), from an in-depth analysis of patient and healthcare provider perspectives on what mobility changes constitutes mild, moderate and severe disability. Design In this prospective, cross-sectional study, a 19-item Disability Questionnaire was developed following literature and expert review. Between 2011 and 2012, the Disability Questionnaire was provided to healthcare providers experienced in CMT attending national scientific meetings, and to patients self-registered with the Inherited Neuropathy Consortium—Rare Diseases Clinical Research Consortium on-line contact registry. Provider and patient responses were compared utilising a two-sided unpaired t test with Bonferroni correction. The questionnaire was then assessed for validity, reliability and unidimensionality. Results We analysed 259 Disability Questionnaires (167 patients, 92 providers); these showed perfect agreement between patient and provider responses on qualitative descriptions of disability, but significant differences in quantitative responses on items corresponding to minimal or severe disability (p<0.001). Validity and test–retest reliability of the questionnaire was excellent (Cronbach's α=0.96; intraclass correlation coefficients (ICC)=0.977 (0.951 to 0.993). Exploratory factor analysis and the Mokken Scaling Procedure supported the unidimensionality of the mobility-Disability Severity Index. Conclusions The mobility-Disability Severity Index is a unique instrument, categorising disability from the patient's perspective, and will undergo further cross-validation studies in CMT.