Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.
Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.
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DOI：
10.1016/j.jpeds.2021.10.053
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发表时间：
2022-03
期刊：
影响因子：
--
通讯作者：
COMBINE Study Group
中科院分区：
文献类型：
--
作者：
Miller TL;Schuchard J;Carle AC;Forrest CB;Kappelman MD;COMBINE Study Group
评估PROMIS儿童患者报告的结局指标是否可作为慢性儿科疾病临床试验的有效终点。我们通过多中心、随机、双盲、安慰剂对照的甲氨蝶呤二元治疗临床结果（COMBINE）试验，评估了PROMIS儿科措施的响应性。我们通过评估患有或未经历疾病活动改善的患者报告的预后（PRO）评分的变化，来检查PROMIS儿科措施的变化与疾病活动变化之间的关系。参与者包括来自35个机构的266名患有乳糜泻的儿童和青少年。在随访过程中，参与者在大多数PRO领域表现出改善，临床改善组观察到的效应量最大。维持无类固醇缓解的患者在疼痛干扰、疲劳和IBD症状方面的PRO得分显著降低，积极情绪得分较高。在一项大型、多中心的儿科CD临床试验中，这项研究证明了儿童PROMIS测量疲劳和疼痛干扰作为研究终点的响应性，扩展了越来越多的研究支持使用PROMIS儿童测量作为临床试验中可靠的PRO终点。
To evaluate whether PROMIS pediatric patient-reported outcome measures serve as valid endpoints in a clinical trial of a chronic pediatric illness. We evaluated the responsiveness of PROMIS Pediatric measures collected through the Clinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) trial, a multicenter, randomized, double-blind, placebo-controlled, pragmatic clinical trial in pediatric patients with CD. We examined the relationships between changes in PROMIS pediatric measures and changes in disease activity by evaluating patient-reported outcome (PRO) score changes among patients who did or did not experience improvement in disease activity. Participants included 266 children and adolescents with CD from a total of 35 institutions. Over the course of follow-up, participants showed improvement in most PRO domains, with the largest effect sizes observed for the clinically improved group. Patients who maintained steroid-free remission showed significantly lower PRO scores for Pain Interference, Fatigue and IBD Symptom and higher Positive Affect scores. This study demonstrates the responsiveness of the Pediatric PROMIS measures of Fatigue and Pain Interference as study endpoints in a large, multi-center pragmatic trial in pediatric CD, extending a growing body of research supporting the use of PROMIS Pediatric measures as reliable PRO endpoints for clinical trials.
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影响因子：
3.2
作者：
Hinds, Pamela S.;Nuss, Suzanne L.;DeWalt, Darren A.
通讯作者：
DeWalt, Darren A.
DOI：
10.1002/art.41888
发表时间：
2021-10
期刊：
Arthritis & rheumatology (Hoboken, N.J.)
影响因子：
--
作者：
Kimura Y;Schanberg LE;Tomlinson GA;Riordan ME;Dennos AC;Del Gaizo V;Murphy KL;Weiss PF;Natter MD;Feldman BM;Ringold S;CARRA STOP-JIA Investigators
通讯作者：
CARRA STOP-JIA Investigators
影响因子：
2.7
作者：
Forrest, Christopher B.;Forrest, Kathryn D.;Furth, Susan L.
通讯作者：
Furth, Susan L.
影响因子：
4.9
作者：
Brenner, Erica J.;Long, Millie D.;Kappelman, Michael D.
通讯作者：
Kappelman, Michael D.
影响因子：
3
作者：
Selewski, David T.;Massengill, Susan F.;Gipson, Debbie S.
通讯作者：
Gipson, Debbie S.