J Neurol Neurosurg Psychiatry 78:411-413 doi:10.1136/jnnp.2006.096008
  • Short report

How is disease progress in Friedreich’s ataxia best measured? A study of four rating scales

  1. M C Fahey1,2,
  2. L Corben1,
  3. V Collins1,
  4. A J Churchyard2,
  5. M B Delatycki1
  1. 1Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Parkville, Victoria, Australia
  2. 2Monash Neurology, Monash Medical Centre, Melbourne, Victoria, Australia
  1. Correspondence to:
 Associate Professor Martin Delatycki
 Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Flemington Road, Parkville, Victoria 3052, Australia; martin.delatycki{at}
  • Received 17 April 2006
  • Accepted 17 October 2006
  • Revised 21 July 2006
  • Published Online First 20 October 2006


Background: Friedreich’s ataxia (FRDA), the most common genetic cause of ataxia, is characterised by progressive neurodegeneration and cardiomyopathy. Initial treatments are likely to slow progression rather than reverse morbidity. An appropriate and sensitive scale to measure disease progress is critical to detect the benefit of treatments.

Objective: To compare the Friedreich Ataxia Rating Scale (FARS) with other scales proposed as outcome measures for FRDA.

Methods: 76 participants were assessed with the FARS and the International Cooperative Ataxia Rating Scale (ICARS) and 72 of these participants were also assessed with the Functional Independence Measure and the Modified Barthel Index. 43 participants had repeat measures at an interval of 12 months. Sensitivity and responsiveness were assessed using the effect size for each measure and the sample size required for a placebo-controlled clinical trial.

Results: The FARS showed a high correlation with the other three measures. A significant change in the score over 12 months was detected by the FARS, the International Cooperative Ataxia Rating Scale and the Functional Independence Measure. The FARS had the greatest effect size and requires fewer patients for an equivalently powered study.

Conclusions: Of the scales assessed, the FARS is the best to use in clinical trials of FRDA. This is based on effect size, and power calculations that show that fewer participants are required to demonstrate the same effect of an intervention. Further work is required to develop more sensitive and responsive instruments.


  • Funding: Funding was received through the National Health and Medical Research Council, Australia (MCF and MBD), the Friedreich Ataxia Research Association (Australasia) and the Friedreich Ataxia Research Alliance (USA).

  • Competing interests: None.

  • Published Online First 20 October 2006

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