title: Seeking a measure of clinically meaningful change in ALS.
abstract: We sought to identify a method to assess 'clinically meaningful change' perceived by patients, caregivers and clinical raters in relation to changes in ALSFRS-R scores at three-month intervals. In this five-site study, 81 patient-caregiver dyads were interviewed at baseline, three, and six months to assess changes in ALSFRS-R in relation to perceived occurrence of change, its magnitude and impact. Ratings by patients, caregivers and clinical raters were analyzed over three-month intervals within and between respondent groups. We found that patients, clinical raters, and caregivers agreed about 80% of the time about whether change occurred, and in what direction, on each of three visits. The perceived magnitude of change for the four domains measured by the ALSFRS-R was correlated with ratings of impact within respondent groups and across time. We also found moderate associations between changes in ALSFRS-R domain scores and judgments of symptom impact as rated by patient, caregiver and clinical rater. Independent measures (Quality of Life, Goal Assessment Scaling) showed no consistent correlations with ALSFRS-R change scores. In conclusion, the use of scales to assess the perceived magnitude and impact of change corresponding with the domains of the ALSFRS-R may be a step towards understanding of the clinical meaning of changes in that measure.
authors.affiliation: New York State Psychiatric Institute , New York, NY.
authors.forename: Martin
authors.initials: M
authors.lastname: McElhiney
authors.forename: Judith G
authors.initials: JG
authors.lastname: Rabkin
authors.forename: Raymond
authors.initials: R
authors.lastname: Goetz
authors.forename: Jonathan
authors.initials: J
authors.lastname: Katz
authors.forename: Robert G
authors.initials: RG
authors.lastname: Miller
authors.forename: Dallas A
authors.initials: DA
authors.lastname: Forshew
authors.forename: William
authors.initials: W
authors.lastname: David
authors.forename: Merit
authors.initials: M
authors.lastname: Cudkowicz
authors.forename: Jonathan D
authors.initials: JD
authors.lastname: Glass
authors.forename: Stanley
authors.initials: S
authors.lastname: Appel
authors.forename: Ericka
authors.initials: E
authors.lastname: Simpson
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Mitsumoto
mesh_terms: D000203:Activities of Daily Living; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000690:Amyotrophic Lateral Sclerosis / Q000175:diagnosis* / Q000523:psychology*; D017028:Caregivers / Q000523:psychology*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D011788:Quality of Life / Q000523:psychology*; D012720:Severity of Illness Index; D018709:Statistics, Nonparametric; D016896:Treatment Outcome; D055815:Young Adult
keywords: ALSFRS-R; Clinically meaningful changes; quality of life