P0876The efficacy of biologics and small molecules in the induction of remission in ulcerative proctitis: an international multicentre study

Abstract Background Patients with ulcerative proctitis (UP) are frequently excluded from clinical trials, and there is limited evidence on the effectiveness of advanced therapies in the management of patients with UP. This study evaluated the effectiveness and safety of biologics and small molecules in patients with moderately to severely active UP in a real-world setting. Methods This international, multicenter, retrospective cohort study included adult patients with moderately to severely active UP treated with advanced therapies between 2000 and 2025. Patients with at least 12 weeks of follow-up were eligible. The primary outcome was steroid-free clinical remission (SF-CR) at week 12 (w12) defined as partial Mayo Score (pMS) ≤2 (including a rectal bleeding subscore=0 and a stool frequency subscore≤1) in the absence of corticosteroid therapy for 12 consecutive weeks. Secondary outcomes included changes in bowel urgency (BU), C-reactive protein (CRP), fecal calprotectin (FC), mayo endoscopic score (MES) and Nancy score at w12. Results A total of 114 patients with UP treated with advanced therapies were included (60.5% female, median age of 44.3 ± 14.8 years). Most patients (55.3%) were naïve to advanced therapies, while 44.7% had received ≥1 advanced therapy, including 18.4% with ≥2 such agents. TNFα inhibitors were the most commonly initiated therapies (41.2%; n = 47: 38 infliximab, 7 adalimumab, 2 golimumab), followed by JAK inhibitors (21.9%; n = 25: 11 filgotinib, 9 upadacitinib, and 5 tofacitinib), ustekinumab (21.1%; n = 24), vedolizumab (6.1%; n = 7), etrasimod (6.1%; n = 7), and mirikizumab (3.5%; n = 4). Concomitant corticosteroids were used in 30.7% of patients. Overall significant reduction in pMS was observed at week 12 (p 0.001, 95% CI 0.97–0.99, n = 108). SF-CR at week 12 was achieved by 68/114 patients (59.6%) (Figure 1). Overall BU improved significantly (p 0.001, 95% CI 0.75–0.94, n = 56). Both FC (p 0.001, 95% CI 0.61–0.84, n = 83) and CRP (p 0.01, 95% CI 0.25–0.62, n = 104) decreased significantly. Endoscopic activity improved, with significant reductions in MES (p 0.01, 95% CI 0.66–0.91, n = 35; 20% achieved MES 0, 34.3% MES 1) and Nancy score (p 0.01, 95% CI 0.35–0.83, n = 30). Overall, six treatment discontinuations were recorded (four for lack of efficacy, one for disease progression, one for an allergic reaction), with no additional safety signals observed. Conclusion This multicentre international study demonstrates the effectiveness and safety of advanced therapy in inducing clinical remission in patients with ulcerative proctitis in a real-life setting. Conflict of interest: D’Amico, Ferdinando: Grant: ECCO fellowship grant 2020 ECCO grant 2021 Personal Fees: F D’Amico has served as a speaker for Abbvie, Alfasigma, Ferring, Lilly, Sandoz, Janssen, Fresenius Kabi, Galapagos, Giuliani, MSD, Pfizer, Takeda, Tillotts, and Omega Pharma he also served as an advisory board member for Abbvie, AnaptysBio, Ferring, Fresenius Kabi, Galapagos, Janssen, Lilly, MSD, Takeda, and Nestlè. This study was conducted as a collaboration between Materia Prima and Pfizer. Materia Prima is the study sponsor Dr. Fanizzi, Fabrizio: No conflict of interest Honap, Sailish: Sailish Honap has served as a speaker, consultant, advisory board member, and/or received travel grants from AbbVie, AlfaSigma, Banook Group, Ferring, Johnson & Johnson, Lilly, Pharmacosmos, Pfizer, and Takeda. Magro, Fernando: Fernando Magro served as speaker and received honoraria from Abbvie, Arena, Biogen, Bristol-Myers Squibb, Falk, Ferring, Hospira, Janssen, Laboratórios Vitoria, Pfizer, Lilly, Merck Sharp & Dohme, Sandoz, Takeda, UCB, Vifor. Ferraz de Oliveira, Paula: No conflict of interest Papa, Alfredo: No conflict of interest Gutiérrez Casbas, Ana: No conflict of interest Kotze, Paulo Gustavo: Grant: Pfizer, Takeda Personal Fees: Abbvie, Johnson & Johnson, Ferring, Pfizer, Takeda, Celltrion Other: Abbvie, Janssen, Ferring, Pfizer, Takeda Armuzzi, Alessandro: Consulting fees from AbbVie, Abivax, Alfa Sigma, Astra Zeneca, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb, Celltrion, Eli-Lilly, Enthera, Ferring, Galapagos, Gilead, Giuliani, Janssen, Lionhealth, MSD, Nestlé, Pfizer, Protagonist Therapeutics, Roche, Samsung Bioepis, Sanofi, Sandoz, Takeda, Teva Pharmaceuticals, Tillots Pharma Speaker’s fees from AbbVie, Abivax, AG Pharma, Alfa Sigma, Biogen, Bristol-Myers Squibb, Celltrion, Eli-Lilly, Ferring, Galapagos, Gilead, Janssen, Lionhealth, MSD, Novartis, Pfizer, Roche, Samsung Bioepis, Sandoz, Takeda, Teva Pharmaceuticals Research support from Biogen, MSD, Takeda, and Pfizer Non-financial support from Abbvie, Janssen, MSD, Pfizer, Takeda Dignass, Axel: Personal Fees: AbbVie, Alfasigma, CED Service GmbH, Celltrion, Dr. Falk Pharma, Falk Foundation, Ferring, Fresenius Kabi, Gilead, High5MD, J & J, Lilly, Materia Prima, MSD, Pfizer, Pharmacosmos, Sandoz, Stada, Streamed-Up, Takeda, Tillotts, Vifor Pharma Grant Abbvie, J & J, Takeda Non-financial support Abbvie, J & J, Takeda Other: Abivax, AbbVie, Alfasigma, Dr Falk Pharma, Fewrring, J & J, Pfizer Kobayashi, Taku: Grant: AbbVie, Alfresa Pharma, Bristol Myers Squibb, Celtrion, EA Pharma, Gilead Sciences, Kyorin Pharmaceutical, Miyarisan, Mochida Pharmaceutical, Nippon Kayaku, Otsuka Holdings, Pfizer, Sekisui Medical, Takeda, Zeria Pharmaceutical Personal Fees: AbbVie, Alfresa Pharma, Alimentiv, Bristol Myers Squibb, Celltrion, Covidien, EA Pharma, Eli Lilly, Ferring Pharmaceuticals, Galapagos, Gilead Sciences, Janssen, JIMRO, Kissei Pharmaceutical, Kyorin Pharmaceutical, Mitsubishi Tanabe Pharma, Mochida Pharmaceutical, MSD, Nippon Kayaku, Pfizer, Sekisui Medical, Takeda Pharmaceutical, Zeria Pharmaceutical Dotan, Iris: Grant: The Leona M. and Harry B. Helmsley Charitable Trust, Altman Research, Pfizer, BMS Personal Fees: Pfizer, Falk, Ferring, Abbvie, Janssen, Celltrion, Takeda, Celgene/BMS, Gilead, Galapagos, Materia Prima, Sandoz, Sublimity, Sangamo, Spyre, Eli-Lilly, Harp Diagnostics, Gutreat, Astra Zeneca Hart, Ailsa: Grant: Takeda Personal Fees: Abbvie, Amgen, Arena, AZ, Falk, Celltrion, Eli Lilly, Ferring, Genentech/ Roche, GSK, Pfizer, Takeda, Napp, Pharmacosmos, Janssen (J & J), Bristol-Myers Squibb, Gilead, Galapagos, Alfasigma Ghosh, Subrata: No conflict of interest Iacucci, Marietta: Grant: Pentax, Olympus, Eli lilly, Helmsley Personal Fees: Pentax, Pfitzer, Janssen, EliLilly, J & J Massimino, Luca: No conflict of interest Jairath, Vipul: Consulting Fees: Abbvie, Alimentiv, Amgen, Anaptys Bio, Asahi Kasei, Asieris, Astra Zeneca, Attovia, Blackbird Labs, BMS, Boehringer Ingleheim, Biomebank, Caldera, Calluna, Catalytic Health, Celltrion, Ensho, Enthera, Exeliome Biosciences, Ferring, Fresenius Kabi, Gilead, Granite Bio, GSK, Janssen, Lilly, Merck, Mountainfield, MRM Health, Nxera, Organon, OSE Immunotherapeutics, Pendopharm, Pioneering Medicine, Pfizer, Prometheus, Roche/Genentech, Sanofi, SCOPE, Shattuck Labs, Sorriso, Spyre, Synedgen, Takeda, Teva, Tillotts, Union Therapeutics, Ventus, Ventyx, Vividion, Xencor, Zealand Pharma. Peyrin-Biroulet, Laurent: CONSULTING Abbvie, Abivax, Adacyte, Alimentiv, Alfasigma, Amgen, Apini, Banook, BMS, Celltrion, Enthera, Ferring, Fresenius Kabi, Galapagos, Genentech, Gilead, Iterative Health, Janssen, Lilly, LifeMine, Medac, Morphic, MSD, Nordic Pharma, Novartis, Oncodesign Precision Medicine, ONO Pharma, OSE Immunotherapeuthics, Par’ Immune, Pfizer, Prometheus, Roche, Roivant, Samsung, Sandoz, Sanofi, Sorriso, Spyre, Takeda, Teva, ThirtyfiveBio, Tillots, Vectivbio, Vedanta, Ventyx. LECTURE Abbvie, Alfasigma, Amgen, Biogen, Celltrion, Ferring, Galapagos, Genentech, Gilead, Iterative Health, Janssen, Lilly, Medac, MSD, Nordic Pharma, Pfizer, Sandoz, Takeda, Tillots Danese, Silvio: Personal Fees: AbbVie, Alimentiv, Allergan, Amgen, Applied Molecular Transport, AstraZeneca, Athos Therapeutics, Biogen, Boehringer Ingelheim, Bristol Myers Squibb, Celgene, Celltrion, Dr Falk Pharma, Eli Lilly, Enthera, Ferring Pharmaceuticals Inc., Gilead, Hospira, Inotrem, Janssen, Johnson & Johnson, Morphic, MSD, Mundipharma, Mylan, Pfizer, Roche, Sandoz, Sublimity Therapeutics, Takeda, Teladoc Health, TiGenix, UCB Inc., Vial, Vifor Lecture fees from Abbvie, Amgen, Ferring Pharmaceuticals Inc., Gilead, Janssen, Mylan, Pfizer, Takeda. This study was conducted as a collaboration between Materia Prima and Pfizer. Materia Prima is the study sponsor