Responsiveness of different rating instruments in spinocerebellar ataxia patients

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 Warrenburg, L. Baliko, B. Melegh, C. DepondtJ. Schulz, S. Tezenas Du Montcel, T. Klockgether

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)678-684
Number of pages7
Issue number8
Publication statusPublished - Feb 2010

ASJC Scopus subject areas

  • Clinical Neurology


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