Effectiveness and safety of ustekinumab in ulcerative colitis: Real-world evidence from the ENEIDA registry

Chaparro, María; Garre, Ana; Iborra, Marisa; Sierra, Mónica; Barreiro-de Acosta, Manuel; Fernández-Clotet, Agnés; de Castro, Luisa; Boscá-Watts, Maia; Casanova, María José; López-García, Alicia; Lorente, Rufo; Rodríguez, Cristina; Carbajo, Ana Y; Arroyo, Maria Teresa; Gutiérrez, Ana; Hinojosa, Joaquín; Martínez-Pérez, Teresa; Villoria, Albert; Bermejo, Fernando; Busquets, David; Camps, Blau; Cañete, Fiorella; Manceñido, Noemí; Monfort, David; Navarro-Llavat, Mercè; Pérez-Calle, José Lázaro; Ramos, Laura; Rivero, Montserrat; Angueira, Teresa; Camo, Patricia; Carpio, Daniel; García-de-la-Filia, Irene; González-Muñoza, Carlos; Hernández, Luis; Huguet, José M; Morales, Víctor J; Sicilia, Beatriz; Vega, Pablo; Vera, Isabel; Zabana, Yamile; Nos, Pilar; Suárez Álvarez, Patricia; Calviño-Suarez, Cristina; Ricart, Elena; Hernández, Vicent; Mínguez, Miguel; Márquez, Lucía; Hervías Cruz, Daniel; Rubio Iturria, Saioa; Barrio, Jesús; Gargayo-Puyuelo, Carla; Francés, Rubén; Hinojosa, Esther; del Moral, María; Calvet, Xavier; Algaba, Alicia; Aldeguer, Xavier; Guardiola, Jordi; Mañosa, Miriam; Pajares, Ramón; Piqueras, Marta; García-Bosch, Orlando; Lopez Serrano, Pilar; Castro, Beatriz; Lucendo, Alfredo J; Montoro, Miguel; Castro Ortiz, Elena; Mesonero, Francisco; García-Planella, Esther; Fuentes, David A; Bort, Inmaculada; Delgado-Guillena, Pedro; Arias, Lara; Iglesias, Agueda; Calvo, Marta; Esteve, Maria; Domènech, Eugeni; Gisbert, Javier P

BACKGROUND: The development program (UNIFI) has shown promising results of ustekinumab in ulcerative colitis (UC) treatment that should be confirmed in clinical practice. AIMS: To evaluate the durability, effectiveness and safety of ustekinumab in UC in real-life. METHODS: Patients included in the prospectively maintained ENEIDA registry who received at least one intravenous dose of ustekinumab due to active UC [Partial Mayo Score (PMS) >2] were included. Clinical activity and effectiveness were defined based on PMS. Short-term response was assessed at week 16. RESULTS: A total of 95 patients were included. At week 16, 53% of patients had response (including 35% of patients in remission). In the multivariate analysis, elevated serum C-reactive protein was the only variable significantly associated with lower likelihood of achieving remission. Remission was achieved in 39% and 33% of patients at weeks 24 and 52, respectively. Thirty-six percent of patients discontinued the treatment with ustekinumab during a median follow-up of 31 weeks. The probability of maintaining ustekinumab treatment was 87% at week 16, 63% at week 56, and 59% at week 72; primary failure was the main reason for ustekinumab discontinuation. No variable was associated with risk of discontinuation. Three patients reported adverse events; one of them had a fatal severe SARS-CoV-2 infection. CONCLUSIONS: Ustekinumab is effective both in the short and the long-term in real-life, even in a highly refractory cohort. Higher inflammatory burden at baseline correlated with lower probability of achieving remission. Safety was consistent with the known profile of ustekinumab.