دورية أكاديمية
أعرض في EDS

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

التفاصيل البيبلوغرافية
العنوان: Timed walk as primary outcome measure of treatment response in clinical trials for HTLV-1-associated myelopathy: a feasibility study.
المؤلفون: Martin F; Department of Biology, Centre for Immunology and Infection, Hull York Medical School, University of York, York, UK., Inoue E; National Centre for Child Health and Development, National Medical Centre for Children and Mothers, Research Institute, Tokyo, Japan., Cortese IC; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, USA., de Almeida Kruschewsky R; Advanced Laboratory of Public Health, Gonçalo Moniz Center, Fundação Oswaldo Cruz, Salvador, Bahia Brazil ; Bahian School of Medicine and Public Health (EBMSP), Salvador, Bahia Brazil., Adonis A; Department of Medicine, Section of Virology, Imperial College London, London, UK., Grassi MF; Advanced Laboratory of Public Health, Gonçalo Moniz Center, Fundação Oswaldo Cruz, Salvador, Bahia Brazil ; Bahian School of Medicine and Public Health (EBMSP), Salvador, Bahia Brazil., Galvão-Castro B; Advanced Laboratory of Public Health, Gonçalo Moniz Center, Fundação Oswaldo Cruz, Salvador, Bahia Brazil ; Bahian School of Medicine and Public Health (EBMSP), Salvador, Bahia Brazil., Jacobson S; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, USA., Yamano Y; Department of Rare Diseases Research, Institute of Medical Science, St. Marianna University Graduate School of Medicine, Kawasaki, Japan., Taylor GP; Department of Medicine, Section of Virology, Imperial College London, London, UK., Bland M; Department of Health Sciences, University of York, York, UK.
المصدر: Pilot and feasibility studies [Pilot Feasibility Stud] 2015 Oct 23; Vol. 1, pp. 35. Date of Electronic Publication: 2015 Oct 23 (Print Publication: 2015).
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: BioMed Central Country of Publication: England NLM ID: 101676536 Publication Model: eCollection Cited Medium: Print ISSN: 2055-5784 (Print) Linking ISSN: 20555784 NLM ISO Abbreviation: Pilot Feasibility Stud Subsets: PubMed not MEDLINE
أسماء مطبوعة: Original Publication: London : BioMed Central, [2015]-
مستخلص: 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.
References: AIDS Res Hum Retroviruses. 2006 Oct;22(10):931-5. (PMID: 17067261)
AIDS Rev. 2011 Jul-Sep;13(3):161-70. (PMID: 21799534)
Neurology. 1996 Apr;46(4):1016-21. (PMID: 8780082)
Control Clin Trials. 1998 Dec;19(6):589-601. (PMID: 9875838)
Proc Natl Acad Sci U S A. 1980 Dec;77(12):7415-9. (PMID: 6261256)
Arch Phys Med Rehabil. 2005 Feb;86(2):190-6. (PMID: 15706542)
J Neurol Neurosurg Psychiatry. 2010 Dec;81(12 ):1336-40. (PMID: 20660921)
Neurology. 2002 Apr 23;58(8):1294-6. (PMID: 11971105)
Arch Phys Med Rehabil. 2001 Jan;82(1):9-13. (PMID: 11239279)
Arch Phys Med Rehabil. 1999 Apr;80(4):442-7. (PMID: 10206608)
Retrovirology. 2006 Sep 19;3:63. (PMID: 16984654)
J Neurol Neurosurg Psychiatry. 2002 Mar;72(3):361-5. (PMID: 11861697)
Phys Ther. 1997 Jan;77(1):19-27. (PMID: 8996460)
Age Ageing. 1997 Jan;26(1):15-9. (PMID: 9143432)
Lancet. 1985 Aug 24;2(8452):407-10. (PMID: 2863442)
فهرسة مساهمة: Keywords: 10-metre walk test; Clinical trial; HAM/TSP; HTLV; Myelopathy; Rare disease; Sample size
تواريخ الأحداث: Date Created: 20161215 Latest Revision: 20200929
رمز التحديث: 20231215
مُعرف محوري في PubMed: PMC5153909
DOI: 10.1186/s40814-015-0031-1
PMID: 27965813
قاعدة البيانات: MEDLINE
الوصف
تدمد:2055-5784
DOI:10.1186/s40814-015-0031-1