Incorporating Patient Perspectives into a Composite Score for Measuring Disease Progression in Spinocerebellar Ataxia (SCA)
This study took a novel approach to enhance the face validity of SCACOMS by incorporating patient feedback into the statistically optimized item weights. The result is the merging of objectively derived item weightings (reflecting optimal scale responsiveness) with patient-assigned relevance. While this update may increase the patient centricity of a composite measure, this comes at the expense of reduced sensitivity. This potential trade-off in sensitivity to detect change should be evaluated in the context of the composite measure's intended use.
Authors
- Suzanne Hendrix (ORCID: https://orcid.org/0000-0002-2374-7727)
- Melissa Beiner
- Ellen Korol
- Sam Dickson
- Vlad Coric
- Susan Perlman
- Basia Rogula (ORCID: https://orcid.org/0000-0002-2115-8275)
- Michele Potashman (ORCID: https://orcid.org/0000-0001-7389-3354)
- Pfizer
- Maggie Heinrich
- G. LʼItalien
- Jeremy D. Schmahmann
- Liana S. Rosenthal
- Linda Abetz-Webb
- Lauren C. Powell
Institutions
- Johns Hopkins University (US)
- University of California, Los Angeles (US)
- PAREXEL International (United Kingdom) (GB)
- Johns Hopkins Medicine (US)
- Patient-Centered Outcomes Research Institute (US)
- Massachusetts General Hospital (US)
- Biohaven Pharmaceuticals (United States) (US)
- Johns Hopkins Hospital (US)
- TetraLogic Pharmaceuticals (United States) (US)
Publication Details
- Journal
- Neurology and Therapy
- Published
- 2026-02-18
- DOI
- https://doi.org/10.1007/s40120-026-00891-6
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00