Author | Martin, Fabiola | |
Author | Inoue, Eisuke | |
Author | Cortese, Irene C. M | |
Author | Kruschewsky, Ramon de Almeida | |
Author | Adonis, Adine | |
Author | Grassi, Maria Fernanda Rios | |
Author | Castro Filho, Bernardo Galvão | |
Author | Jacobson, Steven | |
Author | Yamano, Yoshihisa | |
Author | Taylor, Graham P | |
Author | Bland, Martin | |
Access date | 2016-04-20T17:22:15Z | |
Available date | 2016-04-20T17:22:15Z | |
Document date | 2015 | |
Citation | MARTIN, F. et al. Timed walk as primary outcome measure of treatment response in clinical trials for HTLV-1- associated myelopathy: a feasibility study. Martin et al. Pilot and Feasibility Studies, v. 1, p. 35, 2015. | pt_BR |
ISSN | 2055-5784 | |
URI | https://www.arca.fiocruz.br/handle/icict/13949 | |
Language | eng | pt_BR |
Publisher | BioMed Central | pt_BR |
Rights | open access | pt_BR |
Title | Timed walk as primary outcome measure of treatment response in clinical trials for HTLV-1- associated myelopathy: a feasibility study | pt_BR |
Type | Article | pt_BR |
DOI | 10.1186/s40814-015-0031-1 | |
Abstract | 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 | pt_BR |
Affilliation | University of York. Centre for Immunology and Infection. Department of Biology. Hull York Medical School, York, UK | pt_BR |
Affilliation | National Centre for Child Health and Development. National Medical Centre for Children and Mothers. Research Institute. Tokyo, Japan | pt_BR |
Affilliation | National Institutes of Health. National Institute of Neurological Disorders and Stroke. Bethesda, USA | pt_BR |
Affilliation | Fundação Oswaldo Cruz. Centro de Pesquisas Gonçalo Moniz. Salvador, BA, Brasil / Bahian School of Medicine and Public Health. EBMSP. Salvador, BA, Brasil | pt_BR |
Affilliation | Imperial College London. Department of Medicine. Section of Virology. London, UK | pt_BR |
Affilliation | Fundação Oswaldo Cruz. Centro de Pesquisas Gonçalo Moniz. Salvador, BA, Brasil / Bahian School of Medicine and Public Health. EBMSP. Salvador, BA, Brasil | pt_BR |
Affilliation | Fundação Oswaldo Cruz. Centro de Pesquisas Gonçalo Moniz. Salvador, BA, Brasil / Bahian School of Medicine and Public Health. EBMSP. Salvador, BA, Brasil | pt_BR |
Affilliation | National Institutes of Health. National Institute of Neurological Disorders and Stroke. Bethesda, USA | pt_BR |
Affilliation | Institute of Medical Science, St. Marianna University Graduate School of Medicine. Department of Rare Diseases Research. Kawasaki, Japan. | pt_BR |
Affilliation | National Institutes of Health. National Institute of Neurological Disorders and Stroke. Bethesda, USA | pt_BR |
Affilliation | University of York. Department of Health Sciences. York, UK | pt_BR |
Subject | HTLV | pt_BR |
Subject | Myelopathy | pt_BR |
Subject | HAM/TSP | pt_BR |
Subject | 10-metre walk test | pt_BR |
Subject | Sample size | pt_BR |
Subject | Clinical trial | pt_BR |
Subject | Rare disease | pt_BR |