The Validation of Pediatric Patient Reported Outcomes in Chronic Diseases (PEPR) Consortium (U19) will capitalize on recent advances in the science of Patient Reported Outcomes (PROs) to assess the health of children with a variety of chronic diseases and conditions in clinical research and care settings.
Examples of chronic diseases or conditions in children include (but are not limited to): asthma, juvenile arthritis, cystic fibrosis, diabetes, obesity and overweight, chronic kidney disease, inflammatory bowel disease, sickle cell disease, malnutrition, developmental disabilities including attention deficit/hyperactivity disorder and autism spectrum disorder, cerebral palsy and mental illnesses.
Many of these diseases and conditions cause patients to have pain or experience fatigue; affect their sleep; alter their ability to physically or mentally function on a daily basis; or in other ways interfere with their social relationships and overall quality-of-life. The impact, importance, and value of pediatric self-reporting (and parent-proxy reporting) in the evaluation of treatment efficacy and effectiveness support the need for a universally applicable, common metric in order to allow clinically meaningful comparisons of treatments of disease, both within and across chronic pediatric diseases and conditions.
The PEPR Consortium will feature the utilization of the Patient Reported Outcomes Measurement Information System (PROMIS®) pediatric self-report and parent proxy instruments. These instruments include those already, or soon to be, available at the Assessment Center.
The main, long-term goal of the Consortium is to conduct robust clinical validation studies of child patient-reported outcomes (cPROs) in clinical trials and care settings, and to examine the impact of environmental stressors (including socioeconomic aspects) on children’s symptoms and quality of life in a variety of chronic diseases or conditions. Therefore, cPROs may advance personalized, precision medicine by coupling with detailed clinical phenotyping and/or biospecimen collections in established, well-characterized pediatric cohorts. Development of new domains that extend the measurement science of cPROs to address environmental stressors/exposures, and that fit into the PROMIS domain framework following PROMIS standards, is also possible. Successful applicants will be recognized leaders in clinical pediatric research and patient care with demonstrated expertise in leading large clinical collaborative projects, as well as recruiting and retaining cohort(s) of high quality.
The figure below highlights key features of the structure of the proposed Consortium. Details are included in the FOA.
For NIAMS contacts listed in the FOA, please submit questions to PEPR@mail.nih.gov. In addition to the NIAMS contacts listed in the FOA, the following NIH Institutes are participating in this initiative and the individuals below may be contacted for questions or more information.