Timed walk as primary outcome measure of treatment response in clinical trials for HTLV-1- associated myelopathy: a feasibility study

dc.creatorMartin, Fabiola
dc.creatorInoue, Eisuke
dc.creatorCortese, Irene C. M
dc.creatorKruschewsky, Ramon de Almeida
dc.creatorAdonis, Adine
dc.creatorGrassi, Maria Fernanda Rios
dc.creatorCastro Filho, Bernardo Galvão
dc.creatorJacobson, Steven
dc.creatorYamano, Yoshihisa
dc.creatorTaylor, Graham P
dc.creatorBland, Martin
dc.date2016-04-20T17:22:15Z
dc.date2016-04-20T17:22:15Z
dc.date2015
dc.date.accessioned2023-09-27T00:16:05Z
dc.date.available2023-09-27T00:16:05Z
dc.identifierMARTIN, 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.
dc.identifier2055-5784
dc.identifierhttps://www.arca.fiocruz.br/handle/icict/13949
dc.identifier10.1186/s40814-015-0031-1
dc.identifier.urihttps://repositorioslatinoamericanos.uchile.cl/handle/2250/8899129
dc.descriptionBackground: 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
dc.formatapplication/pdf
dc.languageeng
dc.publisherBioMed Central
dc.rightsopen access
dc.subjectHTLV
dc.subjectMyelopathy
dc.subjectHAM/TSP
dc.subject10-metre walk test
dc.subjectSample size
dc.subjectClinical trial
dc.subjectRare disease
dc.titleTimed walk as primary outcome measure of treatment response in clinical trials for HTLV-1- associated myelopathy: a feasibility study
dc.typeArticle


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