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 journalArticle

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Abstract

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
JournalNeurology
Volume74
Issue number8
DOIs
Publication statusPublished - Feb 2010

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Spinocerebellar Ataxias
Ataxia
Visual Analog Scale
Equipment and Supplies
Diagnostic Self Evaluation
Natural History
Sample Size
Health Status
Disease Progression
Cohort Studies

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Schmitz-Hübsch, T., Fimmers, R., Rakowicz, M., Rola, R., Zdzienicka, E., Fancellu, R., ... Klockgether, T. (2010). Responsiveness of different rating instruments in spinocerebellar ataxia patients. Neurology, 74(8), 678-684. https://doi.org/10.1212/WNL.0b013e3181d1a6c9

Responsiveness of different rating instruments in spinocerebellar ataxia patients. / Schmitz-Hübsch, T.; Fimmers, R.; Rakowicz, M.; Rola, R.; Zdzienicka, E.; Fancellu, R.; Mariotti, C.; Linnemann, C.; Schöls, L.; Timmann, D.; Filla, A.; Salvatore, E.; Infante, J.; Giunti, P.; Labrum, R.; Kremer, B.; Van De Warrenburg, B. P C; Baliko, L.; Melegh, B.; Depondt, C.; Schulz, J.; Du Montcel, S. Tezenas; Klockgether, T.

In: Neurology, Vol. 74, No. 8, 02.2010, p. 678-684.

Research output: Contribution to journalArticle

Schmitz-Hübsch, T, Fimmers, R, Rakowicz, M, Rola, R, Zdzienicka, E, Fancellu, R, Mariotti, C, Linnemann, C, Schöls, L, Timmann, D, Filla, A, Salvatore, E, Infante, J, Giunti, P, Labrum, R, Kremer, B, Van De Warrenburg, BPC, Baliko, L, Melegh, B, Depondt, C, Schulz, J, Du Montcel, ST & Klockgether, T 2010, 'Responsiveness of different rating instruments in spinocerebellar ataxia patients', Neurology, vol. 74, no. 8, pp. 678-684. https://doi.org/10.1212/WNL.0b013e3181d1a6c9
Schmitz-Hübsch T, Fimmers R, Rakowicz M, Rola R, Zdzienicka E, Fancellu R et al. Responsiveness of different rating instruments in spinocerebellar ataxia patients. Neurology. 2010 Feb;74(8):678-684. https://doi.org/10.1212/WNL.0b013e3181d1a6c9
Schmitz-Hübsch, T. ; Fimmers, R. ; Rakowicz, M. ; Rola, R. ; Zdzienicka, E. ; Fancellu, R. ; Mariotti, C. ; Linnemann, C. ; Schöls, L. ; Timmann, D. ; Filla, A. ; Salvatore, E. ; Infante, J. ; Giunti, P. ; Labrum, R. ; Kremer, B. ; Van De Warrenburg, B. P C ; Baliko, L. ; Melegh, B. ; Depondt, C. ; Schulz, J. ; Du Montcel, S. Tezenas ; Klockgether, T. / Responsiveness of different rating instruments in spinocerebellar ataxia patients. In: Neurology. 2010 ; Vol. 74, No. 8. pp. 678-684.
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AU - Schmitz-Hübsch, T.

AU - Fimmers, R.

AU - Rakowicz, M.

AU - Rola, R.

AU - Zdzienicka, E.

AU - Fancellu, R.

AU - Mariotti, C.

AU - Linnemann, C.

AU - Schöls, L.

AU - Timmann, D.

AU - Filla, A.

AU - Salvatore, E.

AU - Infante, J.

AU - Giunti, P.

AU - Labrum, R.

AU - Kremer, B.

AU - Van De Warrenburg, B. P C

AU - Baliko, L.

AU - Melegh, B.

AU - Depondt, C.

AU - Schulz, J.

AU - Du Montcel, S. Tezenas

AU - Klockgether, T.

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N2 - 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.

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