期刊
NEUROLOGY
卷 74, 期 8, 页码 678-684出版社
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1212/WNL.0b013e3181d1a6c9
关键词
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资金
- European Union (EU) EUROSCA
- Polish Ministry of Science
- Italian Ministry of Health
- AIFA (Italian Drug Agency)
- Santhera Pharmaceuticals
- DFG
- BMBF
- Volkswagen Foundation
- HSP-Selbsthilfegruppe Deutschland eV
- Bernd Fink Foundation
- German Heredoataxia Foundation
- Ministry of University (MUR)
- Italian Medicine Agency (AIFA)
- Ataxia UK
- Hersenstichting Nederland (Dutch Brain Society)
- Prinses Beatrix Foundation
- Dutch Medical Research Council
- Radboud University Nijmegen
- Hersenstichting Nederland
- Dutch Brain Foundation
- Pfizer Inc
- Fonds National de la Recherche Scientifique (FNRS, Belgium)
- GlaxoSmithKline
- Merz Pharmaceuticals
- LLC
- Lundbeck Inc.
- Takeda Pharmaceutical Company Limited
Objective: To determine the longitudinal metric properties of recently developed clinical assessment tools in spinocerebellar ataxia (SCA). Methods: A subset of 171 patients from the EUROSCA natural history study cohort (43 SCA1, 61 SCA2, 37 SCA3, and 30 SCA6) were examined after 1 year of follow-up. Score changes and effect size indices were calculated for clinical scales (Scale for the Assessment and Rating of Ataxia [SARA], Inventory of Non-Ataxia Symptoms [INAS]), functional tests (SCA Functional Index [SCAFI] and components), and a patient-based scale for subjective health status (EQ-5D visual analogue scale [EQVAS]). Responsiveness was determined in relation to the patient's global impression (PGI) of change and reproducibility described as retest reliability for the stable groups and smallest detectable change. Results: Within the 1-year follow-up period, SARA, INAS, and SCAFI but not EQVAS indicated worsening in the whole group and in the groups with subjective (PGI) worsening. SCAFI and its 9-hole pegboard (9HPT) component also deteriorated in the stable groups. Standardized response means were highest for 9HPT (-0.67), SARA (0.50), and SCAFI (-0.48) with accordingly lower sample size estimates of 143, 250, or 275 per group for a 2-arm interventional trial that aims to reduce disease progression by 50%. SARA and EQVAS performed best to distinguish groups classified as worse by PGI. All scales except EQVAS reached the criterion for retest reliability. Conclusion: While both the Scale for the Assessment and Rating of Ataxia and the SCA Functional Index (SCAFI) (and its 9-hole pegboard component) had favorable measurement precision, the clinical relevance of SCAFI and 9-hole pegboard score changes warrants further exploration. The EQ-5D visual analogue scale proved insufficient for longitudinal assessment, but validly reflected patients' impression of change. Neurology(R) 2010; 74: 678-684
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