Crohn’s disease (CD) and ulcerative colitis (UC) are chronic inflammatory bowel diseases (IBDs) that affect approximately 1.2 million individuals in the United States, cost over $6 billion annually, and cause substantial patient morbidity, missed work and school, and diminished quality of life. Pediatric onset IBD (PIBD) is a distinct entity, often presenting with more severe and extensive disease, profoundly impacting gastrointestinal health, psychosocial functioning, pubertal development and growth. Since less than 10% of patients with IBD are children, PIBD is recognized as a rare disease by the NIH Office of Rare Diseases Research.
Currently, anti-TNF therapy is considered first line treatment for children with moderate to severe IBD and these treatments are the only FDA-approved biologic agents. However, primary non-response and secondary loss of response are common. In a large, PCORI-funded pragmatic trial recently completed by our team, we observed that approximately 30% of children initiating anti-TNF therapy experienced treatment failure within the first two years.
The overarching goal of this proposal is to leverage PCORnet® to generate real-world evidence regarding the clinical and comparative effectiveness and safety of off-label medications in anti-TNF refractory patients with pediatric IBD through a prospective observational cohort study.