Development of a novel observer reported outcome tool as the primary efficacy outcome measure for a rare disease randomized controlled trial.

开发了一种新型观察者报告的结果工具 ， 作为罕见疾病随机对照试验的主要疗效结果指标。

• 影响指数: 4.534
• 发表时间: 09 2018
• 来源期刊: Mitochondrion
• DOI: 10.1016/j.mito.2017.11.003
• PMID: 29129554
• 文章类型: Journal Article
• 中科院分区: Q3
• 方向: 生物
• 作者列表: Stacpoole PW,Shuster J,Thompson JLPS,Prather RA,Lawson LA,Zou B,Buchsbaum R,Nixon SJ

关键词: Clinical trial; Dichloroacetate; Observer-reported outcome measure; Pyruvate dehydrogenase complex; Rare disease

Mesh: Child; Child, Preschool; Cross-Over Studies; Dichloroacetic Acid / administration & dosage; Female; Humans; Male; Placebos / administration & dosage; Pyruvate Dehydrogenase Complex Deficiency Disease / drug therapy; Rare Diseases / drug therapy; Symptom Assessment / methods; Treatment Outcome

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成果(ObsRO)调查仪器，用于多中心，安慰剂对照,二氯乙酸盐治疗儿童丙酮酸脱氢酶复合物缺乏症的交叉随机对照试验。该仪器量化了受试者在家中的功能水平，据父母/照顾者报告，他们在提供构成仪器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，它易于使用且全面地捕获受影响儿童的主要临床功能限制，并且父母/照顾者每天需要不到5分钟才能完成。