1147 Long-Term Efficacy of Vedolizumab in Pediatric Inflammatory Bowel Disease

Abstract

INTRODUCTION: Pediatric patients (pts) with inflammatory bowel disease (IBD) who have failed anti-TNF therapy have limited treatment options. Vedolizumab (VDZ) has proven to be safe and effective for adults with refractory IBD. While short-term data (≤30 weeks) of VDZ in pediatric IBD pts are promising, long-term outcomes have not been reported. METHODS: We performed a retrospective study of IBD pts ≤18 years who received VDZ for at least 12 months. Data collection included demographics, disease characteristics, therapy, and adverse events (AEs). Disease activity scores were assessed at baseline, 14 weeks, 6, 12, and 24 months using the short pediatric Crohn’s disease (CD) activity index (shPCDAI) and pediatric ulcerative colitis (UC) activity index (PUCAI). Clinical remission was defined by shPCDAI score less than 15 or PUCAI score less than 10. RESULTS: A total of 29 pediatric IBD pts treated with VDZ since drug approval in 2014 were identified. Baseline demographics were as follows: mean age 14 ± 3.2 years, male 17 (59%), CD 16 (55%), UC 13 (45%). All were previously treated with an anti-TNF agent. Median VDZ treatment duration was 21.3 months (IQR 13.8-39.1). VDZ was discontinued in 5 pts for primary non-response (< 6 months) and in 5 pts for secondary non-response (12-24 months). Efficacy data is presented for 24 pts treated with VDZ for 1 year and 14 pts for 2 years. Significant reductions in shPCDAI and PUCAI scores compared to baseline were observed at all time points, as shown in Figure 1. Clinical remission was achieved at 1 year in 8 of 13 (62%) CD pts and 5 of 11 (45%) UC pts. Remission was sustained at 2 years in 8 of 9 (89%) CD pts and 3 of 5 (60%) UC pts [Figure 2]. The most commonly reported AEs were headache and fatigue; no drug discontinuations occurred due to AEs. There was one IBD-related ED visit and three IBD-related hospitalizations, with two resulting in surgery. CONCLUSION: Our data suggest that VDZ is effective in achieving clinical remission in pediatric IBD pts with prior anti-TNF failure, with minimal safety concerns. We observed early and sustained remission rates in both UC and CD pts, with CD pts experiencing higher rates than previously reported in literature. Additional studies are warranted to determine the role of VDZ in the treatment of pediatric IBD.