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Limitations of the Unified Multiple System Atrophy Rating Scale as outcome measure for clinical trials and a roadmap for improvement
- Source :
- Clinical Autonomic Research
- Publication Year :
- 2021
- Publisher :
- Springer Science and Business Media LLC, 2021.
-
Abstract
- Purpose The unified multiple system atrophy (MSA) rating scale (UMSARS) was developed almost 20 years ago as a clinical rating scale to capture multiple aspects of the disease. With its widespread use, the shortcomings of the UMSARS as a clinical outcome assessment (COA) have become increasingly apparent. We here summarize the shortcomings of the scale, confirm some of its limitations with data from the Natural History Study of the Synucleinopathies (NHSS), and suggest a framework to develop and validate an improved COA to be used in future clinical trials of disease-modifying drugs in patients with MSA. Methods Expert consensus assessment of the limitations of the UMSARS and recommendations for the development and validation of a novel COA for MSA. We used UMSARS data from the ongoing NHSS (ClinicalTrials.gov: NCT01799915) to showcase some of these limitations. Results The UMSARS in general, and specific items in particular, have limitations to detect change resulting in a ceiling effect. Some items have specific limitations including unclear anchoring descriptions, lack of correlation with disease severity, susceptibility to improve with symptomatic therapies (e.g., orthostatic hypotension, constipation, and bladder dysfunction), and redundancy, among others. Conclusions Because of the limitations of the UMSARS, developing and validating an improved COA is a priority. The time is right for academic MSA clinicians together with industry, professional societies, and patient advocacy groups to develop and validate a new COA.
- Subjects :
- medicine.medical_specialty
Synucleinopathies
Outcome Assessment
Orthostatic
Review Article
Disease
030204 cardiovascular system & hematology
Orphan diseases
Severity of Illness Index
Patient advocacy
Hypotension, Orthostatic
03 medical and health sciences
0302 clinical medicine
Physical medicine and rehabilitation
Atrophy
Rating scale
Outcome Assessment, Health Care
Clinical outcome assessment
Validation
medicine
Humans
Endocrine and Autonomic Systems
business.industry
Multiple System Atrophy
Endpoint
medicine.disease
Health Care
Clinical trial
Scale (social sciences)
Ceiling effect
Neurology (clinical)
Hypotension
business
030217 neurology & neurosurgery
Natural history study
Subjects
Details
- ISSN :
- 16191560 and 09599851
- Volume :
- 31
- Database :
- OpenAIRE
- Journal :
- Clinical Autonomic Research
- Accession number :
- edsair.doi.dedup.....f57979a4a0bc43a1556a0f0143372a08