Spinocerebellar Ataxia Progression Measured with the Patient‐Reported Outcome Measure of Ataxia DOI Open Access
Alister Burt,

Gilbert L’Italien,

Susan Perlman

et al.

Movement Disorders, Journal Year: 2025, Volume and Issue: unknown

Published: March 4, 2025

ABSTRACT Background The Patient‐Reported Outcome Measure of Ataxia (PROM‐Ataxia) has been validated cross‐sectionally but not longitudinally. Objective We aimed to validate PROM‐Ataxia as a measure patient experience disease over time, examine overall and domain‐specific progression, test convergent validity with other clinical outcome assessments (COAs). Methods derived data from 176 patients spinocerebellar ataxia types 1, 2, 3, 6, 7, 8, or 10 in the Clinical Research Consortium for Study Cerebellar at baseline 1 year. classified patients' severity stage (“severity”) according Friedreich's Rating Scale Functional Staging into mild , moderate severe subgroups. Analyses entire cohort by subgroup included internal consistency, sensitivity severity, predictive modeling score changes, correlations COAs: Brief Scale, Assessment Ataxia, Fatigue Severity Cognitive Affective Syndrome scale, EuroQol 5‐Dimension, responsiveness progression. Results exhibited high consistency correlated COAs. Scores demonstrated evolving experience. Progression was sigmoidal, greatest change patients. Compared COAs, captured most change. Mental features worsened fastest patients, physical activities daily living Conclusion is more sensitive than captures evolution year, reveals Studies larger cohorts different diagnoses longer periods may provide insights further enhance care research. © 2025 International Parkinson Movement Disorder Society.

Language: Английский

Spinocerebellar Ataxia Progression Measured with the Patient‐Reported Outcome Measure of Ataxia DOI Open Access
Alister Burt,

Gilbert L’Italien,

Susan Perlman

et al.

Movement Disorders, Journal Year: 2025, Volume and Issue: unknown

Published: March 4, 2025

ABSTRACT Background The Patient‐Reported Outcome Measure of Ataxia (PROM‐Ataxia) has been validated cross‐sectionally but not longitudinally. Objective We aimed to validate PROM‐Ataxia as a measure patient experience disease over time, examine overall and domain‐specific progression, test convergent validity with other clinical outcome assessments (COAs). Methods derived data from 176 patients spinocerebellar ataxia types 1, 2, 3, 6, 7, 8, or 10 in the Clinical Research Consortium for Study Cerebellar at baseline 1 year. classified patients' severity stage (“severity”) according Friedreich's Rating Scale Functional Staging into mild , moderate severe subgroups. Analyses entire cohort by subgroup included internal consistency, sensitivity severity, predictive modeling score changes, correlations COAs: Brief Scale, Assessment Ataxia, Fatigue Severity Cognitive Affective Syndrome scale, EuroQol 5‐Dimension, responsiveness progression. Results exhibited high consistency correlated COAs. Scores demonstrated evolving experience. Progression was sigmoidal, greatest change patients. Compared COAs, captured most change. Mental features worsened fastest patients, physical activities daily living Conclusion is more sensitive than captures evolution year, reveals Studies larger cohorts different diagnoses longer periods may provide insights further enhance care research. © 2025 International Parkinson Movement Disorder Society.

Language: Английский

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