P0831Real-world Outcomes of Ustekinumab, Vedolizumab, and Tumor Necrosis Factor Inhibitors in Very Early Onset Inflammatory Bowel Disease: A Multicentre Cohort Study

Abstract Background Very early-onset inflammatory bowel disease (VEO-IBD), representing cases diagnosed before age 6 years, is increasing in prevalence. Although VEO-IBD often presents as severe, treatment-resistant disease requiring biologic agents, studies comparing effectiveness between biologics like infliximab (IFX), adalimumab (ADL), ustekinumab (UST) and vedolizumab (VDZ) remain limited. Methods We retrospectively analysed patients with VEO-IBD treated for at least a year from 13 institutions in Japan, evaluating clinical course including the effectiveness of biologics such as IFX, ADL, UST and VDZ. Patients with monogenic IBD were excluded. Steroid-free clinical remission (SFCR) and treatment persistence were assessed separately for first-line and second-line or subsequent biologic therapies. Results We studied 101 VEO-IBD patients (56% male; median age, 3.6 years), including 40 with Crohn’s disease, 52 with ulcerative colitis, and 9 with unclassified IBD. Biologics were used in 67 patients. Among patients who received biologic therapy, 90% were initially treated with a TNF inhibitor. Among biologics used as first-line therapy, SFCR rates at 6 and 12 months were 27% and 19% for IFX (n = 46), 43% and 46% for ADL (n = 14), 0% and 40% for VDZ (n = 5), and 100% and 100% for UST (n = 2). First-line persistence rates at 6 and 12 months were 57% and 36% for IFX, 71% and 48% for ADL, 40% and 40% for VDZ, and 100% and 100% for UST. Among biologics used as second-line or subsequent therapies, SFCR rates at 6 and 12 months were 44% and 45% for UST (n = 36), 40% and 36% for VDZ (n = 16), 0% and 11% for ADL (n = 9), and 17% and 0% for IFX (n = 6). Persistence rates for second-line or subsequent therapies were 79% and 79% for UST, 54% and 46% for VDZ, 67% and 33% for ADL, and 17% and 17% for IFX. Persistence among second-line or subsequent therapies appeared higher with UST than with IFX (P 0.0001) and ADL (P = 0.005). No discontinuations due to infusion reactions or other adverse events occurred with UST or VDZ. Conclusion UST and VDZ were effective and well tolerated even when used as second-line or subsequent therapies for VEO-IBD. Conflict of interest: Dr. Nambu, Ryusuke: R. Nambu received speaker’s fees from AbbVie GK., Takeda Pharmaceutical Co. Ltd. and Mitsubishi Tanabe Pharma Corp. Iwama, Itaru: I. Iwama received speaker’s fees from AbbVie GK. and Mitsubishi Tanabe Pharma Corp., Takeda Pharmaceutical Co. Ltd., Miyarisan Pharmaceutical Co., and Medtronic Co. Takeuchi, Ichiro: Speaker’s fees from AbbVie GK, Takeda Pharmaceutical Co., Ltd., and EA Pharma Co., Ltd. Hagiwara, Shin-ichiro: SI. Hagiwara received speaker’s fees from AbbVie GK., Mitsubishi Tanabe Pharma Corp., Takeda Pharmaceutical Co. Ltd. and Alfresa Pharma Co., Ltd. Etani, Yuri: Y. Etani received speaker’s fees from AbbVie GK. and EA Pharma Co., Ltd. Kaji, Emiri: No conflict of interest Yoden, Atsushi: No conflict of interest Kakuta, Fumihiko: No conflict of interest Hoshi, Yusuke: No conflict of interest Tsumura, Naoya: No conflict of interest Mizuochi, Tatsuki: T. Mizuochi received lecture fees from AbbVie GK, Takeda Pharmaceutical Co., Ltd., Miyarisan Pharmaceutical Co., Ltd., EA Pharma Co., Ltd., Eisai Co., Ltd., Kyorin Pharmaceutical Co., Ltd., Mitsubishi Tanabe Pharma Corporation, Nobelpharma Co., Ltd., Sekisui Medical Co., Ltd. and Nippon Kayaku Co., Ltd., and consulting fees from AbbVie GK and Takeda Pharmaceutical Co., Ltd. Kumagai, Hideki: H. Kumagai received lecture fees from AbbVie GK, Takeda Pharmaceutical Co., Ltd., Miyarisan Pharmaceutical Co., Ltd., Otsuka Pharmaceutical Co., Ltd., Toa Shinyaku Co., Ltd., ASKA Pharmaceutical Co., Ltd, and JCR Pharmaceuticals Co., Ltd. Yokoyama, Koji: No conflict of interest Nishizawa, Takuya: No conflict of interest Usami, Masaaki: M. Usami received speaker’s fees from AbbVie GK., and Miyarisan Pharmaceutical Co., Ltd. Takaki, Yugo: Y. Takaki received speaker’s fees from AbbVie GK, Janssen Pharmaceutical K.K., Kyorin Pharmaceutical Co., Ltd., Miyarisan Pharmaceutical Co., Ltd., Mochida Pharmaceutical Co., Ltd., and Takeda Pharmaceutical Co. Ltd. Ebana, Ryo: No conflict of interest Kurasawa, Shingo: No conflict of interest Fujikawa, Hiroki: No conflict of interest Ishige, Takashi: T. Ishige received speaker’s fees from Mitsubishi Tanabe Pharma Corp., EA Pharma Co., Abbvie GK, Janssen Pharmaceutical K.K., Nippon Kayaku Co., Ltd., Alfresa Pharma Co., Ltd., Takeda Pharmaceutical Co. Ltd., JIMRO Co., Ltd., Eisai Co., Ltd., Sandoz K.K. and Miyarisan Pharmaceutical Co., Ltd. Kudo, Takahiro: T. Kudo received speaker’s fees from AbbVie GK, Takeda Pharmaceutical Co., Ltd., Miyarisan Pharmaceutical Co., Ltd., EA Pharma Co., Ltd., Kyorin Pharmaceutical Co., Ltd., Mitsubishi Tanabe Pharma Corporation, Sandoz K.K., Janssen Pharmaceutical K.K., and Mochida pharmaceutical Co., Ltd. Yoshida, Masashi: No conflict of interest Shimizu, Hirotaka: H. Shimizu received speaker’s fees from AbbVie GK., Takeda Pharmaceutical Co. Ltd., EA Pharma Co., Ltd., Miyarisan Pharmaceutical Co., Ltd., and Covidien Japan Inc. Arai, Katsuhiro: K. Arai received research grant and/or speaker’s fee from AbbVie GK, Janssen Pharmaceutical K.K., Takeda Phartaceutical CO., Ltd., EA Pharma Co., Ltd., Eli Lilly Japan K.K., Pfizer Inc., Zeria Pharmaceutical Co., Ltd., Mitsubishi Tanabe Pharma Corp., Kissei Pharmaceutical Co., Ltd., Miyarisan Pharmaceutical Co., Ltd., Kyorin Pharmaceutical Co., Ltd., Mochida pharmaceutical Co., Ltd., and Bristol Myers Squibb Company.