Clinical Validation of PROMIS Measures in a Pediatric Crohns Disease Clinical Trial

PROMIS 措施在儿科克罗恩病临床试验中的临床验证

• 批准号: 9077849
• 负责人: MICHAEL D KAPPELMAN
• 金额: $ 75.31万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至 2021-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9077849
• 关键词: Affect; Anti-Tumor Necrosis Factor Therapy; Centers of Research Excellence; Characteristics; Child; Child Development; Child health care; Childhood; Chronic; Chronically Ill; Classification; Clinical; Clinical Markers; Clinical Trials; Cognitive; Combined Modality Therapy; Copper; Crohn' s disease; Cross-Sectional Studies; Development; Disease; Equation; Fatigue; Funding; Gastroenterologist; Gastrointestinal tract structure; General Population; Growth; Happiness; Hemorrhagic colitis; Individual; Inflammatory; Inflammatory Bowel Diseases; Interview; Link; Masks; Measures; Medical; Methods; Methotrexate; Modeling; Outcome; Outcomes Research; Pain; Pain interference; Parents; Participant; Patient Outcomes Assessments; Patient-Focused Outcomes; Patients; Personal Satisfaction; Phenotype; Placebos; Psychological Stress; Randomized; Recruitment Activity; Reporting; Research; Research Infrastructure; Research Institute; Research Project Grants; Severity of illness; Structural Models; Structure; Symptoms; TNF gene; Techniques; Testing; Ulcerative Colitis; Validation; base; clinical practice; emotional distress; evidence base; experience; improved; instrument; programs; research in practice; response; treatment effect; trial design

Crohn's disease (CD) is a chronic inflammatory disorder that primarily affects the gastrointestinal tract. It causes significant suffering in affected children, who experience bouts of pain, bloody diarrhea, fatigue, emotional distress, and low levels of positive affect. In April 2015, the Patient-Centered Outcomes Research Institute approved funding for a randomized, double-masked, multicenter, pragmatic clinical trial of anti-TNF combination therapy with methotrexate versus monotherapy (anti-TNF with placebo) in pediatric CD. The trial will recruit patients from the offices of pediatric gastroenterologists who participate in ImproveCareNow, a national network (40% of the nation's pediatric gastroenterologists participate) devoted to improving the health of children with inflammatory bowel disease, both CD and ulcerative colitis. Fortuitously, the trial will begin the same month as the proposed project, creating a unique opportunity for evaluating the validity of PROMIS Pediatric measures. The purpose of the proposed research project is to evaluate the content validity, clinical meaning, responsiveness, and longitudinal construct validity of four PROMIS pediatric instruments (pain interference, fatigue, psychological stress experiences, and positive affect) in 319 children with CD. As a panel, these measures characterize a child's level of suffering and happiness. Relieving suffering by reducing disease activity or palliating symptoms in order to promote a child's well-being and happiness is often the primary objective of medical treatment for pediatric chronic conditions. The CD research project will be one of three linked studies within the Child-Centered Outcomes in Practice and Research (COPR) Center's program of research. Each of the studies will use similar measures, methods, and infrastructure to evaluate the clinical validity of PROMIS among children who are chronically ill. Our specific aims are to: (Aim1) Assess the content validity of the PROMIS Pediatric pain interference, fatigue, psychological stress experiences, and positive affect measures for children with Crohn's disease; (Aim 2) Establish clinically meaningful classifications for the PROMIS Pediatric pain interference, fatigue, psychological stress experiences, and positive affect measures for children with Crohn's disease; (Aim 3) Estimate minimal important differences (MIDs) for the PROMIS Pediatric pain interference, fatigue, psychological stress experiences, and positive affect measures in children with Crohn's disease; and, (Aim 4) Evaluate the longitudinal construct validity of PROMIS Pediatric pain interference, fatigue, psychological stress experiences, and positive affect measures by treatment with methotrexate, clinical markers of disease activity, and sociodemographic characteristics for children with Crohn's disease. If our aims are achieved through the clinical validation of the PROMIS instruments in the well phenotyped children in the anti-TNF trial, we will take major steps toward facilitating the use of PROMIS as standard endpoints in clinical trials and in routine clinical practice for pediatric CD.

克罗恩病（CD）是一种慢性炎症性疾病，主要影响胃肠道。它 在受影响的儿童中造成重大痛苦，他们经历了疼痛，血腥腹泻，疲劳， 情绪困扰，积极影响较低。 2015年4月，以患者为中心的结果研究 Institute批准了抗TNF的随机，双掩蔽，多中心的务实临床试验的资金 小儿CD中的甲氨蝶呤与单一疗法（抗TNF）的联合疗法。试验 将从参加ImpriveCarenow的小儿胃肠病学家办公室招募患者 国家网络（全国40％的小儿胃肠病学家参加）致力于改善健康状况 患有炎症性肠病的儿童，包括CD和溃疡性结肠炎。幸运的是，审判将开始 与拟议项目的同一个月，为评估Promis的有效性创造了独特的机会 小儿措施。拟议的研究项目的目的是评估内容有效性，临床 四种Promis小儿仪器的意义，反应能力和纵向结构有效性（疼痛 319名CD儿童的干扰，疲劳，心理压力经历和积极影响）。作为面板， 这些措施是孩子的痛苦和幸福水平。减轻疾病来缓解痛苦 为了促进孩子的幸福和幸福，活动或姑息症状通常是主要的 儿科慢性病的医疗目的。 CD研究项目将是三个 在实践和研究中以儿童为中心的结果（COPR）中心的计划中的研究计划 研究。每项研究都将使用类似的措施，方法和基础设施来评估临床 promis的有效性在长期患病的儿童中。我们的具体目的是：（ AIM1）评估 Promis小儿疼痛干扰，疲劳，心理压力经历和 克罗恩病儿童的积极影响措施； （AIM 2）建立临床意义 Promis小儿疼痛干扰，疲劳，心理压力经历和 克罗恩病儿童的积极影响措施； （目标3）估计最小的重要差异 （MIDS）用于Promis小儿疼痛干扰，疲劳，心理压力经历和积极 影响克罗恩病儿童的措施； （AIM 4）评估的纵向构造有效性 Promis小儿疼痛干扰，疲劳，心理压力经历和积极的影响措施 通过用甲氨蝶呤治疗，疾病活动的临床标记和社会人口统计学特征 克罗恩病的孩子。如果我们的目标是通过Promis的临床验证实现的 在反TNF试验中，表现良好的儿童中的仪器，我们将采取重大步骤来促进 在临床试验和小儿CD的常规临床实践中，将Promis用作标准终点。