Long-Term Effectiveness and Safety of Ustekinumab in Crohn’s Disease: Results from a Large Real-Life Cohort Study
Authors: Mocci Giammarco , Tursi Antonio , Scaldaferri Franco , Napolitano Daniele , Pugliese Daniela , Capobianco Ivan , Bartocci Bianca , Blasi Valentina , Savarino Edoardo V. , Maniero Daria , Redavid Carlo , Lorenzon Greta , Cuomo Antonio , Donnarumma Laura , Gravina Antonietta Gerarda , Pellegrino Raffaele , Bodini Giorgia , Pasta Andrea , Marzo Manuela , Serio Mariaelena , Scarcelli Antonella , Rodinò Stefano , Sebkova Ladislava , Maconi Giovanni , Cataletti Giovanni , Luppino Ileana , Checchin Davide , Ferronato Antonio , Gaiani Federica , Kayali Stefano , Felice Carla , Pranzo Giuseppe , Catarella Domenico , D’Agostino Dario , Di Bartolo Elisabetta , Lombardi Giovanni , Patturelli Marta , Bendia Emanuele , Bolognini Laura , Balducci Daniele , Quatraccioni Claudia , Martini Francesco , Mucherino Caterina , D’Antonio Elvira , Montesano Laura , Vespere Giuliana , Sedda Silvia , D’Onofrio Vittorio , De Luca Leonardo , Spagnuolo Rocco , Luzza Francesco , Fanigliulo Libera , Rocco Giulia , Sacchi Carlotta , Zampaletta Costantino , Grossi Laurino , Lorenzetti Roberto , Aragona Giovanni , Perazzo Patrizia , Forti Giacomo , Allegretta Leonardo , Cazzato Alessia Immacolata , Scorza Stefano , Cortellini Fabio , Capone Pietro , Villani Guido Daniele , Di Fonzo Michela , Iacopini Federico , Tonti Paolo , Neve Viviana , Colucci Raffaele , Elisei Walter , Monterubbianesi Rita , Faggiani Roberto , Pica Roberta , Pagnini Cristiano , Graziani Maria Giovanna , Di Paolo Maria Carla , Onidi Francesca Maria , Saba Francesco , Dore Maria Pina , Satta Paolo Usai , Picchio Marcello , Papa Alfredo 
Published: November 2024

Background: Ustekinumab (UST) is an interleukin-12/interleukin-23 receptor antagonist approved for the treatment of Crohn’s disease (CD). Only limited real-life data on the long-term outcomes of CD patients treated with UST are available. This study assessed UST’s long-term effectiveness and safety in a large population-based cohort of moderate to severe CD patients. Methods: This was a multicenter, retrospective, observational cohort study that included both naïve and biologic-experienced patients treated with UST who achieved clinical remission or clinical response after at least one year of treatment. Clinical activity was scored according to the Harvey–Bradshaw Index (HBI). The primary endpoints were the maintenance or achievement of clinical remission after a further 12-month period of treatment, defined as an HBI of ≤5, and safety. Other endpoints included steroid-free remission, mucosal healing (MH), steroid discontinuation, and the need for treatment optimization during the follow-up. Results: Out of 562 CD patients, after an overall 24-month follow-up, clinical remission was present in 450 (80.0%) patients, and at 12 months, clinical remission was observed in 417/437 (95.4%) patients; 33/125 (26.4%) showed clinical response at 12 months (p = 0.000). A total of 38/103 (36.9%) patients achieved MH. Only 2.1% (12/562), 3% (17/562), and 1.1% (6/562) of patients required surgery, optimization, and re-induction, respectively. Adverse events occurred in eight patients (1.42%). According to a multivariate analysis, the only predictor of long-term remission was the presence of remission at the 12-month follow-up (p = 0.000). Conclusions: Long-term treatment with UST presents good efficacy and safety profiles in CD patients, especially for patients who achieve remission after one year.