Validation of PROMIS Measures in Children with Brain Tumors

描述(申请人提供)：尽管肿瘤和治疗的负面影响已被证明，但脑肿瘤(BT)儿童的健康相关生活质量(QOL)尚未得到很好的研究。儿童BT患者是一个特别具有挑战性的群体，因为他们的患者数量相对较少，疾病的多样性，肿瘤对功能的影响以及手术和治疗效果的范围。目前的提案旨在通过验证儿童BT人群中的PROMIS测量(疲劳、行动不便、上肢、抑郁症状、焦虑和同伴关系)来解决这一问题。将PROMIS措施的使用推广到儿童Bt人群，将提供一个有用的衡量标准，使临床研究中能够评估PROMIS领域和工具的可用性。这将使临床研究人员能够监测治疗的长期效果，并比较不同治疗的有效性，最终导致 改善对这一陷入困境但研究不足的人群的护理。我们计划从两项现有的研究中招募520名患有脑瘤的5-17岁儿童及其父母：儿童肿瘤组第二阶段试验(ACNS1123/ALTE07C1)和质子协作小组注册研究(PCG注册)。所有儿童的父母将完成父母代理版本；8-17岁(预期n=420)的患者将完成儿科PROMIS的自我报告版本。招聘将遵循现有研究的方案，但限于三年，以便目前的研究可以在本RFA允许的项目期限内完成(4年)。我们提出以下具体目标：目标1：验证儿童脑肿瘤的PROMIS措施。具体地说，我们计划a)使用项目反应理论(IRT)模型来评估用于评估脑瘤儿童的儿童PROMIS工具的心理测量学特性；b)评估不同项目的功能，以确保脑瘤人群中的项目参数与美国普通人群相比具有可比性；c)使用已知组、收敛和并发的效度和反应性分析来评估儿童PROMIS工具的有效性；以及d)使用临床锚并经临床医生和健康结果研究人员验证来估计最小重要的临床差异。 目的2：提高儿科PROMIS措施的实用性，为科研和实践服务。具体地说，我们计划a)比较计算机化适应性测试(CAT)和短表格获得的分数的精度水平以及它们对12个月内变化的敏感度；以及b)将两个国家的结果衡量标准标准化努力-PROMIS和神经疾病生活质量(Neuro-QOL)衡量-联系起来。

DESCRIPTION (provided by applicant): Despite the demonstrated negative effects from tumors and treatments, the health-related quality of life (QOL) of children with brain tumor (BT) has not been well studied. Pediatric BT patients are a particularly challenging group to study because of the relatively small number of patients, diversity of disease, varying functional impact of the tumors, and range of surgical and treatment effects. The current proposal aims to address this issue by validating PROMIS measures (Fatigue, Mobility, Upper Extremity, Depressive Symptoms, Anxiety, and Peer Relationships) in the pediatric BT population. Extending the use of PROMIS measures to the pediatric BT population will provide a useful metric that will enable the evaluation of the usability of the PROMIS domains and instruments in clinical studies. This will make it possible for clinical investigators to monitor the long-term effects of treatment and compare the effectiveness of different treatments, ultimately resulting in improved care for this beleaguered yet under-studied population. We plan to recruit 520 children with brain tumors aged 5-17 and their parents from two existing studies: the Children's Oncology Group phase 2 trial (ACNS1123/ ALTE07C1) and the Proton Collaborative Group Registry study (PCG Registry). Parents of all children will complete the parent-proxy version; patients aged 8-17 (anticipated n=420) will complete the self-reported version of the pediatric PROMIS. Recruitment will follow the protocols of the existing studies but be limited to three years so that the current study can be completed within the allowable project length of this RFA (4 years). We propose the following Specific Aims: Aim 1: To validate pediatric PROMIS measures in children with brain tumors. Specifically, we plan to a) evaluate the psychometric properties of pediatric PROMIS measures used to assess children with brain tumors using item response theory (IRT) models; b) evaluate differential item functioning to ensure comparable item parameters within the brain tumor population compared to the US general population; c) evaluate the validity of pediatric PROMIS instruments using known-groups, convergent and concurrent validity and responsiveness analyses; and d) estimate minimally important clinical differences using clinical anchors with verification by clinicians and health outcome researchers. Aim 2: To enhance the utility of the pediatric PROMIS measures for research and practice. Specifically, we plan to a) compare precision levels of scores obtained from Computerized Adaptive Testing (CAT) versus those from short-forms and their sensitivity to changes over 12 months; and b) link comparable outcome areas between two national efforts of standardization of outcome measures - PROMIS and Quality of Life in Neurological Disorders (Neuro-QOL) Measures.