title: Clinical significance in the change of decline in ALSFRS-R.
abstract: Our objective was to survey ALS clinicians and researchers regarding what percentage reduction in the ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised) slope they would consider clinically meaningful. A nine-question survey was provided to 65 members of the Northeast ALS Consortium (NEALS). They were asked to rate the clinical relevance of 10-50% changes in decline of the ALSFRS-R slope on a seven-point scale (1-7), where 1='not at all clinically meaningful', 4='somewhat clinically meaningful', and 7='very clinically meaningful'. Ninety per cent of participants rated a 20% change in the decline of the ALSFRS-R score as the percentage in which a somewhat clinically significant change starts to be noted (i.e. score of 4 or higher). All participants endorsed a 25% or higher change in the ALSFRS-R score as at least somewhat clinically meaningful (score of 4 or higher). Ninety-three per cent of the participants viewed a 50% change in decline as very clinically meaningful (score of 7). This survey demonstrated that the majority of clinicians and clinical researchers surveyed believe that a therapy that resulted in a change of 20% or greater in the slope of the ALSFRS-R would be clinically meaningful.
authors.affiliation: Massachusetts General Hospital, Charlestown, 02129, USA. mcastrillo-viguera@partners.org <mcastrillo-viguera@partners.org>
authors.forename: Carmen
authors.initials: C
authors.lastname: Castrillo-Viguera
authors.forename: Daniela L
authors.initials: DL
authors.lastname: Grasso
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Simpson
authors.forename: Jeremy
authors.initials: J
authors.lastname: Shefner
authors.forename: Merit E
authors.initials: ME
authors.lastname: Cudkowicz
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000175:diagnosis / Q000503:physiopathology*; D004185:Disability Evaluation; D018450:Disease Progression; D019538:Health Care Surveys; D006304:Health Status; D006801:Humans; D009462:Neurology; D017063:Outcome Assessment, Health Care / Q000592:standards; D015203:Reproducibility of Results; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires / Q000592:standards