Effectiveness and Safety of Ustekinumab for Ulcerative Colitis: A Brazilian Multicentric Observational Study

Rogério Serafim Parra; Júlio Maria Fonseca Chebli; Matheus Freitas Cardoso de Azevedo; Liliana Andrade Chebli; Gilmara Pandolfo Zabot; Ornella Sari Cassol; Renata de Sá Brito Fróes; Genoile Oliveira Santana; Márcio Lubini; Daniela Oliveira Magro; Marcello Imbrizi; Antonio Carlos da Silva Moraes; Fabio Vieira Teixeira; Antonio José Tiburcio Alves Junior; Newton Luiz Tricarico Gasparetti Junior; Sandro da Costa Ferreira; Natália Sousa Freitas Queiroz; Paulo Gustavo Kotze; Omar Féres

doi:10.1093/crocol/otae023

Effectiveness and Safety of Ustekinumab for Ulcerative Colitis: A Brazilian Multicentric Observational Study

Crohns Colitis 360. 2024 Apr 11;6(2):otae023. doi: 10.1093/crocol/otae023. eCollection 2024 Apr.

Authors

Rogério Serafim Parra¹, Júlio Maria Fonseca Chebli², Matheus Freitas Cardoso de Azevedo³, Liliana Andrade Chebli², Gilmara Pandolfo Zabot⁴, Ornella Sari Cassol⁵, Renata de Sá Brito Fróes⁶, Genoile Oliveira Santana⁷, Márcio Lubini⁸, Daniela Oliveira Magro⁹, Marcello Imbrizi⁹, Antonio Carlos da Silva Moraes¹⁰, Fabio Vieira Teixeira¹¹, Antonio José Tiburcio Alves Junior¹², Newton Luiz Tricarico Gasparetti Junior¹³, Sandro da Costa Ferreira¹⁴, Natália Sousa Freitas Queiroz^{15

16

10}, Paulo Gustavo Kotze^{15

5}, Omar Féres¹

Affiliations

¹ Department of Surgery and Anatomy, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, São Paulo, Brazil.
² Division of Gastroenterology, Department of Medicine, Inflammatory Bowel Disease Center, Federal University of Juiz de Fora, Juiz de Fora, Minas Gerais, Brazil.
³ Department of Gastroenterology, University of São Paulo School of Medicine, São Paulo, Brazil.
⁴ Department of Colon and Rectum Surgery, Moinhos de Vento Hospital, Feevale University, Porto Alegre, Brazil.
⁵ Department of Colorectal Surgery, Hospital de Clínicas de Passo Fundo, Atitus Medical School, Rio Grande do Sul, Brazil.
⁶ Department of Gastroenterology and Endoscopy, Gastromed, Rio de Janeiro, Brazil.
⁷ Department of Life Sciences, State University of Bahia, Salvador, Bahia, Brazil.
⁸ Department of Surgery, Passo Fundo University, Rio Grande do Sul, Brazil.
⁹ Department of Surgery, State University of Campinas (UNICAMP), Campinas, São Paulo, Brazil.
¹⁰ Internal Medicine Department, Hospital Copa D´Or, Rio de Janeiro, Brazil.
¹¹ Department of Gastroenterology, Gastrosaúde Clinic, Marilia, São Paulo, Brazil.
¹² Department of Surgery, PUC-Campinas Medical School, PUC-Campinas University, Campinas, São Paulo, Brazil.
¹³ Department of Surgery, Hospital Vivalle Rede Dor, São José dos Campos, São Paulo, Brazil.
¹⁴ Department of Medicine, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, São Paulo, Brazil.
¹⁵ Health Sciences Graduate Program, Pontifícia Universidade Católica do Paraná (PUCPR), Curitiba, Brazil.
¹⁶ IBD Center, Santa Cruz Hospital, Curitiba, Brazil.

Abstract

Background: Real-world data on the effectiveness and safety of ustekinumab (UST) in ulcerative colitis (UC) are lacking in Latin America. In this study, we aimed to describe the effectiveness and safety of UST in a real-world multicenter cohort of Brazilian patients with UC.

Methods: We conducted a multicenter retrospective observational cohort study, including patients with moderate-to-severe UC (total Mayo score 6-12, with an endoscopic subscore of 2 or 3) who received UST. The co-primary endpoints were clinical remission, defined as a total Mayo score ≤2 at 1 year, with a combined rectal bleeding and stool frequency subscore of ≤1, and endoscopic remission (endoscopic Mayo subscore of 0) within 1 year from baseline. Secondary endpoints included clinical response between weeks 12 and 16, endoscopic response within 1 year of starting UST, steroid-free clinical remission at week 52, and biochemical remission at week 52. We also evaluated UST treatment persistence and safety.

Results: A total of 50 patients were included (female, n = 36, 72.0%), with a median disease duration of 9.2 years (1-27). Most patients had extensive colitis (n = 38, 76.0%), and 43 (86.0%) were steroid dependent at baseline. Forty patients (80.0%) were previously exposed to biologics (anti-TNF drugs, n = 31; vedolizumab [VDZ], n = 27). The co-primary endpoints of clinical remission at 1 year and endoscopic remission within 1 year were achieved by 50.0% and 36.0% of patients, respectively. Clinical response at weeks 12-16 was 56.0%, and endoscopic response, steroid-free clinical remission, and biochemical remission at week 52 were 68.0%, 46.5%, and 50.0%, respectively. The UST treatment persistence rate at 24 months was 73.7%. During the follow-up, 10 patients (20.0%) were hospitalized, mostly due to disease progression, and 3 patients required colectomy. Nine patients (18.0%) discontinued the drug mainly due to a lack of effectiveness. Twenty-seven adverse events (AEs) were reported, 16 of which were considered as serious AEs.

Conclusions: In this real-world cohort of difficult-to-treat UC patients, UST was associated with improvements in clinical, biochemical, and endoscopic outcomes. The safety profile was favorable, consistent with the known profile of UST.

Keywords: biological therapy; inflammatory bowel disease; real world; ulcerative colitis; ustekinumab.