Advancing the Science of Pediatric Patient-Reported Outcomes for Children with Chronic Disease (PEPR)

Disease Area:
Juvenile Idiopathic Arthritis (JIA)

Biosample Status:
No Biosample

About the PEPR Study

Improving treatment, outcomes, and wellbeing are important to ensuring that young patients growing up with a rheumatic disease live well, feel heard, and are heard. The PEPR study seeks to broaden and deepen our understanding of the social, emotional and physical functioning of children and adolescents living with chronic diseases – including pediatric-onset rheumatic diseases.

Researchers want to learn:

• How patient-reported outcomes (PROs) on social, emotional, and physical functioning compare against clinical measures of their disease activity

Validating measures that matter to patients and parents will provide important, actionable insights so that treatments are best used and their effectiveness is thoughtfully evaluated against a patient’s lived experiences.

Study Updates and Findings

• Health-related quality of life (HRQOL) is multi-dimensional, reflecting the disease itself and factors related to treatment.
• Bad experiences with treatments were associated with lower quality of life among a group of children with JIA, so treatment experience is important to assess routinely.
• Children with chronic conditions, including JIA, report more anxiety, fatigue, pain, and mobility issues than the general pediatric population, so collecting patient-reported outcomes directly from patients is important for complementing information from clinical evaluations.

Read more about the findings:

• Adding patient-reported outcomes to a multisite registry to quantify quality of life and experiences of disease and treatment for youth with juvenile idiopathic arthritis – PMC
• Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases – The Journal of Pediatrics

About the Participants

• Young adults with JIA and SLE. Only children and young adults with official diagnoses of JIA and lupus enrolled in the CARRA Registry.
• Enrolled in the CARRA Registry. Participants must be between 8 and 18 years old at the time of enrollment in the Registry.
• Able to complete study measures on a tablet. Participants and their parents complete patient-reported outcomes on a tablet every six months when they are at their regular CARRA-related clinic visit.

About the Study Team

PEPR is funded by the National Institutes of Health (NIH) and administered by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). The PEPR CARRA study is also part of a larger consortium, the Pediatric Patient Reported Outcomes in Chronic Diseases (PEPR) Consortium.

Lead researchers:

• Elissa Weitzman, ScD, MSc, Boston Children’s Hospital and Harvard Medical School
• Laura Schanberg, MD, Duke University School of Medicine
• Emily von Scheven, MD, MAS, University of California San Francisco
• Sarah Ringold, MD, Seattle Children’s Hospital and the University of Washington School of Medicine (former)

To learn more about this project, please contact [email protected].