Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design
Identifieur interne : 000075 ( Pmc/Corpus ); précédent : 000074; suivant : 000076Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design
Auteurs : Lisa S. Friedman ; Jennifer M. Farmer ; Susan Perlman ; George Wilmot ; Christopher Gomez ; Khalaf O. Bushara ; Katherine D. Mathews ; S. H. Subramony ; Tetsuo Ashizawa ; Laura J. Balcer ; Robert B. Wilson ; David R. LynchSource :
- Movement disorders : official journal of the Movement Disorder Society [ 0885-3185 ] ; 2010.
Abstract
Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia of all four limbs, dysarthria and arreflexia. A variety of measures are currently used to quantify disease progression, including the Friedreich Ataxia Rating Scale, examiner-rated functional disability scales, self-reported activities of daily living and performance measures such as the timed 25-foot walk, 9-hole pegboard test, PATA speech test, and low-contrast letter acuity vision charts. The present study examines the rate of disease progression over one and two years in a cohort of 236 Friedreich ataxia patients using these scales and performance measure composites. The Friedreich Ataxia Rating Scale and performance-measure composites captured disease progression, with a greater sensitivity to change over two years than over one year. The measures differed in their sensitivity to change and in possible bias. These results help to establish norms for progression in FRDA that can be useful in measuring the long-term success of therapeutic agents and defining sample-size calculations for double-blind clinical trials.
Url:
DOI: 10.1002/mds.22912
PubMed: 20063431
PubMed Central: 2954653
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