Comparative Effectiveness of Anti-TNF in Combination with Low Dose Methotrexate vs Anti-TNF Monotherapy in Pediatrics Crohn's Disease (COMBINE), United States, 2015-2022 (ICPSR 38680)
The COMBINE study was a longitudinal examination of pediatric Crohn's Disease (CD) patients in the United States with data collected from 2015-2022. This study was a randomized, double blind, placebo controlled pragmatic trial to compare low dose oral methotrexate versus a placebo in children with Crohn's disease initiating anti-TNF (tumor necrosis factor) therapy with Infliximab or Adalimumab. Eligible participants were randomized with a 1:1 allocation and followed for a minimum of 12 months and maximum of 36 months in the context of routine clinical care. The primary outcome was a composite of indicators of treatment failure and/or toxicity. Secondary outcomes included patient reported outcomes of pain interference and fatigue.
Crohn's disease (CD) is a chronic inflammatory bowel disease (IBD) that affects approximately 600,000 Americans with estimated direct costs of $3.6 billion annually. Typical symptoms (e.g., abdominal pain, bloody diarrhea) result in substantial morbidity, including hospitalization and surgery, missed work and school, and diminished quality of life. The primary treatment goals for all CD patients are to induce remission by eradicating intestinal inflammation and related symptoms and maintain remission by preventing disease flares and progression. Additional treatment goals for pediatric CD include restoring physical and emotional development.
Improving Family-Centered Pediatric Trauma Care: The Standard of Care Versus the Virtual Pediatric Trauma Center, California, 2020-2022 (ICPSR 39210)
Preserving Kidney Function in Children with Chronic Kidney Disease (PRESERVE), United States, 2009-2024 (ICPSR 39689)
The Preserving Kidney Function in Children With Chronic Kidney Disease (PRESERVE) study was designed to provide new knowledge to inform shared decision-making regarding blood pressure (BP) management for pediatric chronic kidney disease (CKD). PRESERVE compared the effectiveness of alternative strategies for monitoring and treating hypertension on preserving kidney function; expanded the National Patient-Centered Clinical Research Network (PCORnet) Common Data Model by adding pediatric- and kidney-specific variables and linking electronic health record data to other kidney disease databases; and assessed the lived experiences of patients related to BP management.
Participants were recruited from 15 clinical institutions across the United States. The research team analyzed electronic health record (EHR) data from 11,851 children with CKD and their caregivers to compare different ways to monitor and treat BP to preserve kidney function. In addition, a subset of patients and caregivers completed an online survey detailing patient-reported outcomes, such as fatigue, life satisfaction, pain levels, sleep disturbance, anxiety, and peer relationships (n=395).
Due to the risk of re-identification based on unique patterns in the individual-level PCORnet electronic health record (EHR) data, patient privacy regulations prohibit the public release of the individual-level data. This collection contains the code underlying the analysis; instructions, codesets, and output lists for the PCORnet queries; and the survey questionnaires for patients and family members.