Clinical measures of disease used in research and healthcare delivery settings do not fully capture the effects of chronic diseases and their treatment burden on children and adolescents. If identified, such factors could be directly addressed through focused comprehensive care of the child and her/his family. Advancing the availability and usefulness of patient reported assessment tools to inform research and clinical care aligns with the federal priority for improving treatment through rigorous comparative effectiveness research and is integral to precision medicine, an enterprise predicated on robust actionable phenotypic patient descriptions. To more fully capture youth's experiences and perspectives, the Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric measures were designed and have undergone extensive psychometric evaluation; however, these instruments continue to be underutilized by the research and clinical communities. The proposed project involves longitudinal clinical validation of PROMIS Pediatric measures undertaken with a large diverse sample of 1500 children/adolescents with rheumatic disease, cancer or inflammatory bowel disease and their parent/caregiver proxies. Participants are engaged through large, extant, multisite clinical and public health research platforms. Participants will provide time series reports using PROMIS measures for validation research. The first objective of this research is to evaluate the responsiveness of the PROMIS Pediatric measures to detect change in health related quality of life (HRQOL) over time and its association with clinical anchors and patient-reported symptom toxicities. Through time series analysis of prospectively engaged pediatric cohorts, we will ascertain whether PROMIS Pediatric measures of psychological stress, depression, anxiety, mobility, fatigue, and pain interference are responsive to prospectively observed changes in treatment and disease activity. The second objective is to evaluate whether PROMIS Pediatric measures of depression and anxiety are associated with subsequent measures of health status including physical and social health, disease-specific outcomes and substance use, after adjusting for relevant measures of baseline health status using a retrospective cohort study of prospectively collected data. The primary exposures of interest will be measured by PROMIS Pediatric depression and anxiety domains. Outcomes will be assessed at one or more subsequent time points to ascertain whether depression and anxiety increase the risk of subsequent disease relapse and other negative health outcomes. Finally, the third objective of this study is to determine the association between steps taken (pedometer data) and PROMIS Pediatric measures of physical activity and function-mobility with exploratory analyses of associations among daily steps, pain interference, fatigue, and depression. Findings from the research project will provide a rich and rigorously evaluated set of pediatric measures for widespread use in clinical research and care to improve efforts to address the full needs of children and adolescents with serious chronic medical conditions.
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