Validation of PROMIS Measures in Children with Brain Tumors

描述（由申请人提供）：尽管肿瘤和治疗的负面影响已得到证实，但脑肿瘤（BT）儿童的健康相关生活质量（QOL）尚未得到充分研究。儿科BT患者是一个特别具有挑战性的研究群体，因为患者数量相对较少，疾病多样性，肿瘤的功能影响不同，手术和治疗效果范围也不同。目前的提案旨在通过验证儿科BT人群的PROMIS指标（疲劳、活动性、上肢功能、抑郁症状、焦虑和同伴关系）来解决这一问题。将PROMIS测量的使用扩展到儿科BT人群将提供一个有用的指标，可以在临床研究中评价PROMIS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 改善对这一被围困但研究不足的人群的护理。我们计划从两项现有研究中招募520名5-17岁的脑肿瘤儿童及其父母：儿童肿瘤组2期试验（ACNS 1123/ALTE 07 C1）和质子协作组登记研究（PCG登记）。所有儿童的父母将完成父母代理版本; 8-17岁的患者（预期n=420）将完成儿科PROMIS的自我报告版本。招募将遵循现有研究的方案，但限于3年，以便在本RFA允许的项目长度（4年）内完成当前研究。我们提出了以下具体目标：目标1：验证儿科PROMIS措施在儿童脑肿瘤。具体而言，我们计划a） 评估使用项目反应理论（IRT）模型评估脑肿瘤儿童的儿科PROMIS测量的心理测量特性; B）评估差异项目功能，以确保 与美国普通人群相比，脑肿瘤人群中的可比项目参数; c）使用已知组、收敛和并行有效性和反应性分析评价儿科PROMIS工具的有效性; d）使用临床锚点估计最小重要临床差异，并由临床医生和健康结局研究人员进行验证。 目的2：提高儿科PROMIS措施的研究和实践的效用。具体而言，我们计划a）比较计算机化自适应测试（CAT）与简表的评分精度水平及其对12个月内变化的敏感性;和B）将两个国家成果标准化措施- PROMIS和神经疾病生活质量（Neuro-QOL）措施之间的可比成果领域联系起来。

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.