Published: Jan. 1, 2025
Language: Английский
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
Citations
1
Neurology and Therapy, Journal Year: 2025, Volume and Issue: unknown
Published: Jan. 13, 2025
The Friedreich Ataxia Rating Scale–Activities of Daily Living (FARS-ADL) is a valid, highly utilized measure for assessing ADL impacts in patients with ataxia. We provide evidence the psychometric validity FARS-ADL two cohorts spinocerebellar ataxia (SCA). Using data from cohort real-world subjects SCA (recruited at Massachusetts General Hospital [MGH]; n = 33) and phase 3 trial troriluzole adults (NCT03701399 [Study 206]; 217), comprising subset SCA3 genotype (n 89), measurement properties minimal change thresholds were examined. Ceiling effects absent within MGH while floor observed eight nine items. Excellent internal consistency reliability was (αtotal 0.88; αitems−removed 0.86–0.87), item-to-total correlations acceptable (r 0.55–0.89 per item). Convergent divergent supported strong demonstrated between scales measuring similar concepts (Neuro-QOL [Upper], Neuro-QOL [Lower], PROM-ADL, PROM-PHYS, FARS-FUNC; all P < 0.001) weaker shown measures differing constructs. A two- to three-point threshold meaningful changes as 0.5 × SD 2.43, SEM 2.19. Mean baseline classified "improved," "no change," or "deteriorated" −0.54, 0.22, 1.47, respectively. Similar trends Study 206 all-SCA cohorts. Psychometric evaluation showed that performed well on analyses examining can detect SCA, including those SCA3. ClinicalTrials.gov identifier, NCT03701399 (Study 206).
Language: Английский
Citations
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The Cerebellum, Journal Year: 2025, Volume and Issue: 24(3)
Published: March 19, 2025
Abstract Multiple sclerosis (MS) is a chronic neurodegenerative disorder often associated with cerebellar ataxia. Accurate assessment of ataxia crucial for monitoring disease progression and guiding rehabilitation. The Scale the Assessment Rating Ataxia (SARA) clinician-reported outcome measure (ClinRO) designed to evaluate severity. This study aimed translate, culturally adapt, validate Italian version SARA in individuals MS. follows cross-sectional design was conducted at Neurorehabilitation Unit Sapienza University Rome. translation followed ISPOR ISOQOL guidelines ensure linguistic cultural equivalence. Psychometric properties evaluation included internal consistency (Cronbach’s alpha), test-retest reliability (intraclass correlation coefficient [ICC]), construct validity (correlation Berg Balance [BBS], Mini-Balance Evaluation Systems Test [Mini-BESTest], Timed Up Go [TUG] test), cross-cultural validity. Seventy-five MS patients (EDSS ≤ 6.5) were recruited. demonstrated excellent alpha = 0.855) (ICC 0.993). Strong negative correlations found BBS ( r -0.838, p < 0.001) Mini-BESTest -0.767, 0.001), supporting Significant differences emerged based on age, employment status, EDSS scores. valid reliable tool assessing severity Its strong psychometric support its use clinical research settings. Future studies should explore responsiveness rehabilitation interventions.
Language: Английский
Citations
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Journal of Neurology, Journal Year: 2025, Volume and Issue: 272(5)
Published: April 10, 2025
Language: Английский
Citations
0
Published: Jan. 1, 2025
Language: Английский
Citations
0