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TIMED WALK AS PRIMARY OUTCOME MEASURE OF TREATMENT RESPONSE IN CLINICAL TRIALS FOR HTLV-1- ASSOCIATED MYELOPATHY: A FEASIBILITY STUDY
Myelopathy
HAM/TSP
10-metre walk test
Sample size
Clinical trial
Rare disease
Autor
Afiliación
University of York. Centre for Immunology and Infection. Department of Biology. Hull York Medical School, York, UK
National Centre for Child Health and Development. National Medical Centre for Children and Mothers. Research Institute. Tokyo, Japan
National Institutes of Health. National Institute of Neurological Disorders and Stroke. Bethesda, USA
Fundação Oswaldo Cruz. Centro de Pesquisas Gonçalo Moniz. Salvador, BA, Brasil / Bahian School of Medicine and Public Health. EBMSP. Salvador, BA, Brasil
Imperial College London. Department of Medicine. Section of Virology. London, UK
Fundação Oswaldo Cruz. Centro de Pesquisas Gonçalo Moniz. Salvador, BA, Brasil / Bahian School of Medicine and Public Health. EBMSP. Salvador, BA, Brasil
Fundação Oswaldo Cruz. Centro de Pesquisas Gonçalo Moniz. Salvador, BA, Brasil / Bahian School of Medicine and Public Health. EBMSP. Salvador, BA, Brasil
National Institutes of Health. National Institute of Neurological Disorders and Stroke. Bethesda, USA
Institute of Medical Science, St. Marianna University Graduate School of Medicine. Department of Rare Diseases Research. Kawasaki, Japan.
National Institutes of Health. National Institute of Neurological Disorders and Stroke. Bethesda, USA
University of York. Department of Health Sciences. York, UK
National Centre for Child Health and Development. National Medical Centre for Children and Mothers. Research Institute. Tokyo, Japan
National Institutes of Health. National Institute of Neurological Disorders and Stroke. Bethesda, USA
Fundação Oswaldo Cruz. Centro de Pesquisas Gonçalo Moniz. Salvador, BA, Brasil / Bahian School of Medicine and Public Health. EBMSP. Salvador, BA, Brasil
Imperial College London. Department of Medicine. Section of Virology. London, UK
Fundação Oswaldo Cruz. Centro de Pesquisas Gonçalo Moniz. Salvador, BA, Brasil / Bahian School of Medicine and Public Health. EBMSP. Salvador, BA, Brasil
Fundação Oswaldo Cruz. Centro de Pesquisas Gonçalo Moniz. Salvador, BA, Brasil / Bahian School of Medicine and Public Health. EBMSP. Salvador, BA, Brasil
National Institutes of Health. National Institute of Neurological Disorders and Stroke. Bethesda, USA
Institute of Medical Science, St. Marianna University Graduate School of Medicine. Department of Rare Diseases Research. Kawasaki, Japan.
National Institutes of Health. National Institute of Neurological Disorders and Stroke. Bethesda, USA
University of York. Department of Health Sciences. York, UK
Resumen en ingles
Background: To advance the treatment of HTLV-1-associated myelopathy/tropical spastic paraparesis (HAM/TSP),
randomised controlled therapeutic studies with appropriate and sensitive outcomes are reuired. One candidate
outcome is the 10-metre walk test (10MWT), a patient-centred, simple and functional measure. To calculate sample size
based on 10MWT as the primary outcome, variability within and between subjects must be known.
Methods: Data on 10MWT from 76 patients with HAM/TSP were prospectively collected from four specialist centres in
Brazil, Japan, USA and UK. Data, collected at two time points, 6 months apart, were log transformed and subjected to
analysis of covariance.
Results: Baseline mean (standard deviation = SD), median 10MWT were 23.5 (18.9), 16.3 s/10 m and at 6 months 24.9
(23.9), 16.4 s/10 m. The mean (SD) % increase in walk time was 5.74 % (28.2 %). After logarithmic transformation, the
linear correlation between baseline and 24 weeks 10MWT was r = 0.938. Using these data, it was determined that a
randomised controlled trial with 30 participants per group would have 90 % power to detect a 19 % decrease or a
23 % increase in 10MWT.
Conclusions: The intra-patient variability of 10MWT is relatively small in HAM/TSP over 6 months. 10MWT is a feasible
outcome measure for a clinical trial in HAM/TSP. To our knowledge, this is the first ever recommendation for the sample
size required for trials in HAM/TSP patients
Palabras clave en ingles
HTLVMyelopathy
HAM/TSP
10-metre walk test
Sample size
Clinical trial
Rare disease
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