title: Activity limitations in patients with neuromuscular disorders: a responsiveness study of the ACTIVLIM questionnaire.
abstract: Recently, a self-reported scale of activity limitations, the ACTIVLIM questionnaire, was developed and validated in patients with neuromuscular disorders (NMD). The purpose of this study was to investigate its sensitivity to change. One hundred thirty-two patients with NMD (mean age, range: 31, 6-80) were assessed twice, with 21+/-4 months in between, using the ACTIVLIM questionnaire. Mean score change, effect size, standardized response, mean paired t-test and an individual-level statistical approach were calculated for groups of patients according to their self-rated functional status evolution and for three main diagnostic groups (Ambulant and wheelchair-bound Duchenne muscular dystrophy patients, myotonic dystrophy patients, and patients with Charcot-Marie-Tooth neuropathy). The responsiveness indices showed that the change in activity measures was higher in patients who reported deteriorated functional status and in patients with Duchenne muscular dystrophy. The ACTIVLIM questionnaire showed a good sensitivity to change and could be useful in research settings to characterize the disease course of NMD.
authors.affiliation: Rehabilitation and Physical Medicine unit, Université catholique de Louvain, Unité de Réadaptation et de Médecine Physique (READ 5375), Tour Pasteur, 53, Av Mounier, 1200 Brussels, Belgium.
authors.forename: Laure
authors.initials: L
authors.lastname: Vandervelde
authors.forename: Peter Y K
authors.initials: PY
authors.lastname: Van den Bergh
authors.forename: Nathalie
authors.initials: N
authors.lastname: Goemans
authors.forename: Jean-Louis
authors.initials: JL
authors.lastname: Thonnard
mesh_terms: D000203:Activities of Daily Living; D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002607:Charcot-Marie-Tooth Disease / Q000453:epidemiology; D002648:Child; D004185:Disability Evaluation; D018450:Disease Progression; D005260:Female; D020233:Gait Disorders, Neurologic / Q000175:diagnosis* / Q000453:epidemiology; D006801:Humans; D008297:Male; D008875:Middle Aged; D018908:Muscle Weakness / Q000175:diagnosis* / Q000453:epidemiology; D020388:Muscular Dystrophy, Duchenne / Q000453:epidemiology; D009223:Myotonic Dystrophy / Q000453:epidemiology; D009468:Neuromuscular Diseases / Q000175:diagnosis* / Q000453:epidemiology; D010291:Paresis / Q000175:diagnosis* / Q000453:epidemiology; D011237:Predictive Value of Tests; D011795:Surveys and Questionnaires; D055815:Young Adult