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.

Key words::

• Clinically meaningful changes
• ALSFRS-R
• quality of life

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Correction to: ‘Seeking a measure of clinically meaningful change in ALS’

Acknowledgements

The authors acknowledge the following who were instrumental in carrying out this project: Allison Marziliano and Jonathan Hupf at Columbia, Padma Yerramilli-Rao at Massachusetts General, William Harris at California Pacific Medical Center, Latoya Shaw at Emory, and Sharon Halton and Luis Lay Jr. at Methodist Neurological Institute. We would also like to thank the patients and caregivers who generously gave their time to participate in the interviews.

Declaration of interest: The authors report no conflicts of interest. The authors alone are responsible for the content and writing of the paper.