开发一种新型观察者报告结局工具作为罕见病随机对照试验的主要疗效结局测量指标。
Development of a novel observer reported outcome tool as the primary efficacy outcome measure for a rare disease randomized controlled trial.
机构信息
Department of Medicine (Division of Endocrinology, Diabetes and Metabolism) and Biochemistry and Molecular Biology, University of Florida, College of Medicine, Gainesville, FL 32610, United States.
Department of Health Outcomes and Policy, University of Florida, College of Medicine, Gainesville, FL 32610, United States.
出版信息
Mitochondrion. 2018 Sep;42:59-63. doi: 10.1016/j.mito.2017.11.003. Epub 2017 Nov 10.
Abstract
We developed an Observer-Reported Outcome (ObsRO) survey instrument to be applied in a multicenter, placebo-controlled, crossover randomized controlled trial of dichloroacetate in children with pyruvate dehydrogenase complex deficiency. The instrument quantifies a subject's at-home level of functionality, as reported by a parent/caregiver, who were instrumental in providing the clinical descriptors and domains that formed the instrument's content. Feasibility testing of the ObsRO tool showed it to be easy to use and comprehensive in capturing the major clinical functional limitations of affected children and requires less than 5min for a parent/caregiver to complete daily.
摘要
我们开发了一种观察者报告结局（ObsRO）调查工具，将在一项多中心、安慰剂对照、交叉随机对照试验中应用于二氯乙酸盐治疗丙酮酸脱氢酶复合物缺陷儿童。该工具通过家长/护理人员的报告量化了受试者在家中的功能水平，他们在提供形成工具内容的临床描述符和领域方面发挥了重要作用。ObsRO 工具的可行性测试表明，它易于使用，能够全面捕捉受影响儿童的主要临床功能限制，并且家长/护理人员每天完成的时间不到 5 分钟。
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