A Single-Center Study of Long-Term Effectiveness of Vedolizumab in Anti-TNF Refractory Pediatric Inflammatory Bowel Disease.

Vedolizumab is an anti-α4β7 integrin antibody that has been used successfully in the treatment of adult-onset inflammatory bowel diseases (IBDs: Crohn disease [CD] and ulcerative colitis [UC]). Its off-label use in the pediatric IBD (PIBD) population is increasing, but knowledge on durability beyond 6 months of treatment is limited. Methods: A real-life, single-center, retrospective study of PIBD patients treated with vedolizumab was performed. Data on demographics, prior and concomitant treatments, and disease activity were obtained at 14 weeks, 26 weeks, 1 year, and 2 years of therapy. Primary outcome was corticosteroid- and other biologic-free remission (based on pediatric ulcerative colitis activity index [PUCAI]). Results: Thirty-nine patients were studied. By 1 year, 65% of CD and 68% of UC patients continued on vedolizumab therapy. Corticosteroid- and other biologic-free remission was 29% in CD and 16% in UC. By 2 years, 36% of CD and 47% of UC patients continued therapy. Corticosteroid- and other biologic-free remission was 21% in CD and 40% in UC. By 2 years, 80% of CD and 100% of UC patients were on intensified treatment regimen compared to the manufacturer guidance. Nine patients (23%) required surgical intervention within 26 months of starting vedolizumab indicating the severity of IBD in this cohort. Conclusions: Vedolizumab is a useful therapeutic modality in PIBD patients refractory to anti-TNF therapy, although with declining effectiveness by 2 years. Intensified treatment regimens are associated with long-term durability. Larger prospective trials in children are warranted.