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.

National Institute of Health (NIH)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Research Program--Cooperative Agreements (U19)
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Special Emphasis Panel (ZAR1-KM (M2))
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University of North Carolina Chapel Hill
Chapel Hill
United States
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Nigrovic, Peter A; Beukelman, Timothy; Tomlinson, George et al. (2018) Bayesian comparative effectiveness study of four consensus treatment plans for initial management of systemic juvenile idiopathic arthritis: FiRst-Line Options for Systemic juvenile idiopathic arthritis Treatment (FROST). Clin Trials 15:268-277
Balevic, Stephen J; Becker, Mara L; Cohen-Wolkowiez, Michael et al. (2017) Clinical Trial Design in Juvenile Idiopathic Arthritis. Paediatr Drugs 19:379-389
Hersh, Aimee O; Salimian, Parissa K; Weitzman, Elissa R (2016) Using Patient-Reported Outcome Measures to Capture the Patient's Voice in Research and Care of Juvenile Idiopathic Arthritis. Rheum Dis Clin North Am 42:333-46