Responsiveness of different rating instruments in spinocerebellar ataxia patients

• DOI: 10.1212/wnl.0b013e3181d1a6c9
• Publication Date: 2010-02-22T22:50:43Z
• Authors: T. Schmitz-Hübsch; R. Fimmers; M. Rakowicz; R. Rola; E. Zdzienicka; R. Fancellu; C. Mariotti; C. Linnemann; L. Schöls; D. Timmann; A. Filla; E. Salvatore; J. Infante; P. Giunti; R. Labrum; B. Kremer; B.P.C. van de War...; L. Baliko; B. Melegh; C. Depondt; J. Schulz; S. Tezenas du Mon...; T. Klockgether
• Keywords: Natural history study

<b>Objective:</b> To determine the longitudinal metric properties of recently developed clinical assessment tools in spinocerebellar ataxia (SCA). <b>Methods:</b> A subset 171 patients from EUROSCA natural history study cohort (43 SCA1, 61 SCA2, 37 SCA3, and 30 SCA6) were examined after 1 year follow-up. Score changes effect size indices calculated for scales (Scale Assessment Rating Ataxia [SARA], Inventory Non-Ataxia Symptoms [INAS]), functional tests (SCA Functional Index [SCAFI] components), a patient-based scale subjective health status (EQ-5D visual analogue [EQVAS]). Responsiveness was determined relation to patient9s global impression (PGI) change reproducibility described as retest reliability stable groups smallest detectable change. <b>Results:</b> Within 1-year follow-up period, SARA, INAS, SCAFI but not EQVAS indicated worsening whole group with worsening. its 9-hole pegboard (9HPT) component also deteriorated groups. Standardized response means highest 9HPT (−0.67), SARA (0.50), (−0.48) accordingly lower sample estimates 143, 250, or 275 per 2-arm interventional trial that aims reduce disease progression by 50%. performed best distinguish classified worse PGI. All except reached criterion reliability. <b>Conclusion:</b> While both Scale SCA (SCAFI) (and component) had favorable measurement precision, relevance score warrants further exploration. The EQ-5D proved insufficient assessment, validly reflected patients9