Enhancing Meaningfulness and Usefulness of Pediatric and Caregiver PROMIS Measures Across Illness Groups

项目2摘要/摘要 一个患病儿童的个体疾病经历不能仅仅通过生理参数来预测， 是生理和社会心理因素之间复杂的相互作用的结果。儿童和青少年 相同的疾病有不同的结果和症状。改善每个儿童的健康状况 因此，需要关注他们疾病经历的所有方面-生理、情感和社会。 PROMIS措施代表了一套强大的问卷调查，用于捕获患者的经验。然而，在这方面， 在将其用于临床试验或将其引入临床护理之前， 必须获得意义。目前的项目是一个更大的研究计划的第二部分， 验证PROMIS儿科措施并评估临床意义。项目1将产生强大的 通过一项纵向队列研究获得的风湿性疾病、癌症和IBD儿童的临床和PRO数据 超过1500名8-20岁的患者。本项目的总体目标是利用来自 项目1，以构建可用于临床实践的临床医生仪表板的必要组件。我们 我们将探索利益相关者的需求，并利用先进的分析方法来提高临床 PROMIS儿科措施的意义，通过利用强大的临床表型，纵向 队列研究提供。这三个具体目标是：1）生成允许预测 使用PROMIS测量的重要临床结局。这将通过使用潜在轮廓分析来实现 而不是允许呈现个体患者标准回归和建模方法 在整个人口的背景下。2)计算最小重要差异（MID） PROMIS测量，因此可以对患者评分的变化进行临床解释。3）确定切割- 在与包括青少年、父母和其他利益攸关方在内的主要利益攸关方举行的讲习班上使用标准制定方法， 每个疾病组的临床医生。该项目利用现有登记系统识别受试者， 数据收集和数据管理。它涉及三个疾病组的PROMIS措施， 让不同的利益相关者-患者、家长和提供者-参与定性方法。验证和 PROMIS测量的临床意义的确定将使未来的研究者能够利用这些方法 在他们的临床试验中采取措施，以更好地评估干预措施对儿童疾病经历的影响。 此外，通过该项目，我们将开发实用临床工具的必要组件， 将PROMIS措施与临床环境中使用的解释性指南相结合。下一步将是 在临床实践中实施这些工具，并对这些工具的影响进行广泛评估 临床决策和病人治疗结果的影响

Project 2 Abstract/Summary An ill child's individual disease experience cannot be predicted by physiologic parameters alone, but rather results from a complex interaction between physiologic and psychosocial factors. Children and adolescents with identical diseases have unique outcomes and symptoms. Improving the health outcome of every child thus necessitates attending to all aspects of their disease experience-physiologic, emotional, and social. PROMIS measures represent a robust set of questionnaires for capturing the patient's experience. However, before using them in clinical trials or introducing them into clinical care, information about their clinical meaningfulness must be obtained. The current project represents part 2 of a larger research initiative aimed at validating PROMIS Pediatric measures and assessing clinical meaningfulness. Project 1 will generate robust clinical and PRO data on children with rheumatic disease, cancer and IBD, through a longitudinal cohort study of over 1500 patients ages 8-20 years. The overall goal of the current project is to utilize the information from project 1 to build the necessary components for a clinician dashboard that can be used in clinical practice. We will explore the needs of our stakeholders and utilize advanced analytic approaches to enhance clinical meaning for the PROMIS Pediatric measures by exploiting the robust clinical phenotyping that the longitudinal cohort study provides. The three specific aims are to 1) Generate patient profiles that allow for the prediction of important clinical outcomes using the PROMIS measures. This will be achieved by using latent profile analysis instead of standard regression and modeling approaches, which allow for presentation of individual patient results in the context of the entire population. 2) Calculate minimally important differences (MID) for the PROMIS measures, so that changes in patient scores can be clinically interpreted. And 3) Determine cut- points using standard setting methods in workshops with key stakeholders including adolescents, parents, and clinicians in each disease group. This project leverages existing registries for identification of subjects and for data collection and data management. It addresses PROMIS measures across three disease groups, and engages diverse stakeholders- patients, parents and providers, for the qualitative methods. The validation and ascertainment of clinical meaning of the PROMIS measures will position future investigators to utilize these measures in their clinical trials to better evaluate the effect of interventions on the child's disease experience. Additionally, through this project we will develop the necessary components for practical clinical tools that integrate PROMIS measures with interpretative guidelines for use in the clinical setting. The next steps will be to implement the tools in clinical practice, and to conduct a wide-scale assessment of the impact of these tools on clinical decision-making and patient outcomes.