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dc.contributor.authorChen, Tai-Hengen_US
dc.contributor.authorYang, Yi-Hsinen_US
dc.contributor.authorMai, Hui-Hsiaen_US
dc.contributor.authorLiang, Wen-Chenen_US
dc.contributor.authorWu, Yi-Chingen_US
dc.contributor.authorWang, Hui-Yien_US
dc.contributor.authorJong, Yuh-Jyhen_US
dc.date.accessioned2015-07-21T08:28:19Z-
dc.date.available2015-07-21T08:28:19Z-
dc.date.issued2014-12-01en_US
dc.identifier.issn0883-0738en_US
dc.identifier.urihttp://dx.doi.org/10.1177/0883073813506935en_US
dc.identifier.urihttp://hdl.handle.net/11536/124088-
dc.description.abstractWe used at-home assessments in a clinical trial to relieve the visit burden for participants. A total of 57 patients with type II or III spinal muscular atrophy were enrolled and 10 of them (7 type II and 3 type III) received at-home assessments. The primary end points were Gross Motor Function Measure, Manual Muscle Test, and serum biomarker. The secondary endpoints were Modified Hammersmith Functional Motor Scale and forced vital capacity. The correlation coefficients and analysis of covariance showed good reliability and validity of all outcome measures. Except for Gross Motor Function Measure and Modified Hammersmith Functional Motor Scale, there were no significant differences in measures between in-hospital and at-home groups (intersubject) or among 3 patients who received both at-home and in-hospital visits (intrasubject). We concluded that at-home assessments could provide sufficient reliability in a controlled trial. This modification could help design a successful clinical trial for spinal muscular atrophy.en_US
dc.language.isoen_USen_US
dc.subjectspinal muscular atrophyen_US
dc.subjectclinical trialen_US
dc.subjectoutcome measuresen_US
dc.subjectreliability and validityen_US
dc.titleReliability and Validity of Outcome Measures of In-Hospital and At-Home Visits in a Randomized, Double-Blind, Placebo-Controlled Trial for Spinal Muscular Atrophyen_US
dc.typeArticleen_US
dc.identifier.doi10.1177/0883073813506935en_US
dc.identifier.journalJOURNAL OF CHILD NEUROLOGYen_US
dc.citation.volume29en_US
dc.citation.issue12en_US
dc.citation.spage1680en_US
dc.citation.epage1684en_US
dc.contributor.department生物科技學系zh_TW
dc.contributor.departmentDepartment of Biological Science and Technologyen_US
dc.identifier.wosnumberWOS:000346908000022en_US
dc.citation.woscount0en_US
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