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Limitations of the Unified Multiple System Atrophy Rating Scale as outcome measure for clinical trials and a roadmap for improvement

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dc.contributor.author Palma, José Alberto
dc.contributor.author Millar Vernetti, Patricio
dc.contributor.author Perez, Miguel A.
dc.contributor.author Krismer, Florian
dc.contributor.author Seppi, Klaus
dc.contributor.author Fanciulli, Alessandra
dc.contributor.author Singer, Wolfgang
dc.contributor.author Low, Phillip
dc.contributor.author Biaggioni, Italo
dc.contributor.author Norcliffe-Kaufmann, Lucy
dc.contributor.author Pellecchia, Maria Teresa
dc.contributor.author Martí, Maria José
dc.contributor.author Kim, Han-Joon
dc.contributor.author Merello, Marcelo
dc.contributor.author Stankovic, Iva
dc.contributor.author Poewe, Werner
dc.contributor.author Betensky, Rebecca
dc.contributor.author Wenning, Gregor
dc.contributor.author Kaufmann, Horacio
dc.date.accessioned 2021-02-22T13:37:54Z
dc.date.available 2021-02-22T13:37:54Z
dc.date.issued 2021-02-07
dc.identifier.citation Palma JA, Millar Vernetti P, Perez MA, Krismer F, Seppi K, Fanciulli A, Singer W, Low P, Biaggioni I, Norcliffe-Kaufmann L, Pellecchia MT, Martí MJ, Kim HJ, Merello M, Stankovic I, Poewe W, Betensky R, Wenning G, Kaufmann H. Limitations of the Unified Multiple System Atrophy Rating Scale as outcome measure for clinical trials and a roadmap for improvement. Clin Auton Res. 2021 Feb 7:1–8. doi: 10.1007/s10286-021-00782-w. Epub ahead of print. https://pubmed.ncbi.nlm.nih.gov/33554315/ en_US
dc.identifier.uri https://doi.org/10.1007/s10286-021-00782-w
dc.identifier.uri https://repositorio.fleni.org.ar/handle/123456789/363
dc.description.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. en_US
dc.language.iso eng en_US
dc.publisher Springer en_US
dc.rights info:eu-repo/semantics/openAccess
dc.rights.uri https://creativecommons.org/licenses/by/2.5/ar/
dc.subject Synucleinopathies en_US
dc.subject Sinucleinopatías en_US
dc.subject Outcome Assessment, Health Care en_US
dc.subject Evaluación de Resultado en la Atención de Salud en_US
dc.subject Rare Diseases en_US
dc.subject Enfermedades Raras en_US
dc.title Limitations of the Unified Multiple System Atrophy Rating Scale as outcome measure for clinical trials and a roadmap for improvement en_US
dc.type info:eu-repo/semantics/publishedVersion
dc.type info:eu-repo/semantics/article en_US
dc.description.fil Fil: Palma, José Alberto. New York University School of Medicine; Estados Unidos.
dc.description.fil Fil: Millar Vernetti, Patricio. New York University School of Medicine; Estados Unidos.
dc.description.fil Fil: Perez, Miguel A. New York University School of Medicine; Estados Unidos.
dc.description.fil Fil: Krismer, Florian. Medical University of Innsbruck; Austria.
dc.description.fil Fil: Seppi, Klaus. Medical University of Innsbruck; Austria.
dc.description.fil Fil: Fanciulli, Alessandra. Medical University of Innsbruck; Austria.
dc.description.fil Fil: Singer, Wolfgang. Mayo Clinic Rochester; Estados Unidos.
dc.description.fil Fil: Low, Phillip. Mayo Clinic Rochester; Estados Unidos.
dc.description.fil Fil: Biaggioni, Italo. Vanderbilt University; Estados Unidos.
dc.description.fil Fil: Norcliffe-Kaufmann, Lucy. New York University School of Medicine; Estados Unidos.
dc.description.fil Fil: Pellecchia, Maria Teresa. Salerno University; Italia.
dc.description.fil Fil: Martí, Maria José. Hospital Clinic Barcelona; España.
dc.description.fil Fil: Kim, Han-Joon. Seoul Medical University; Corea del Sur.
dc.description.fil Fil: Merello, Marcelo. Fleni. Departamento de Neurología. Servicio de Movimientos Anormales; Argentina.
dc.description.fil Fil: Stankovic, Iva. University of Belgrade; Serbia.
dc.description.fil Fil: Poewe, Werner. Medical University of Innsbruck; Austria.
dc.description.fil Fil: Betensky, Rebecca. New York University School of Global Public Health; Estados Unidos.
dc.description.fil Fil: Wenning, Gregor. Medical University of Innsbruck; Austria.
dc.description.fil Fil: Kaufmann, Horacio. New York University School of Medicine; Estados Unidos.
dc.relation.ispartofCOUNTRY Alemania
dc.relation.ispartofCITY Darnstadt
dc.relation.ispartofTITLE Clinical autonomic research
dc.relation.ispartofISSN 1619-1560
dc.type.snrd info:ar-repo/semantics/artículo es_ES


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