Novel Biological Agents for Paediatric Inflammatory Bowel Disease at A Tertiary Children's Hospital in China

Objective Vedolizumab and ustekinumab as novel biological agents can be used as second-line biologic agents in children with refractory inflammatory bowel disease. Vedolizumab is a humanized monoclonal antibody that can specifically antagonize α4β7 integrin to prevent the migration and infiltration of inflammatory cells to the gut. Ustekinumab is a human monoclonal antibody targeting interleukins 12 and 23 to reduces intestinal inflammation through a downregulation of IL-12 signaling pathways. This study aimed to evaluate efficacy and safety of novel biological agents in children with with refractory inflammatory bowel disease. Methods We retrospectively analyzed the medical records of IBD patients aged under 18 years treated with VZD or UST at a pediatric IBD center in China to evaluate efficacy and safety. The primary outcome was the steroid-free clinical remission rate at weeks 14 and 52 in VDZ group, while the UST group was evaluated at week 24. The steroid-free remission rate beyond week 52 was also evaluated. Disease activity index and endoscopic score were used to assess disease activity. Results Twenty patients were included (male:female = 10:10, UC:CD = 9:11). The corticosteroid-free clinical remission rates in VDZ group at week 14 and week 52 were 88.9% and 100%, respectively. The corticosteroid-free clinical remission rates in UST group at week 24 and last follow-up were both 100%. The endoscopic remission rates in VDZ group at week 14 and week 52 were 43% and 40%, respectively. Four (50% of those in whom endoscopy was performed) of UST patients achieved endoscopic remission. No adverse events were recorded during the study period. Conclusion Vedolizumab and Ustekinumab, as novel biological agents, may be an effective and safe treatment option for pediatric inflammatory bowel disease patients having nonresponse or adverse reactions to anti-tumor necrosis factor agents.