Seeking a measure of clinically meaningful change in ALS

Martin Mcelhiney, Judith G. Rabkin, Raymond Goetz, Jonathan Katz, Robert G. Miller, Dallas A. Forshew, William David, Merit Cudkowicz, Jonathan D. Glass, Stanley Appel, Ericka Simpson, Hiroshi Mitsumoto

Research output: Contribution to journalArticlepeer-review

13 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)398-405
Number of pages8
JournalAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Issue number5-6
StatePublished - Sep 2014


  • Clinically meaningful changes
  • Quality of life

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology


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