Effectiveness and Safety of Ustekinumab for Moderate to Severely Active Crohn's Disease: Results from an Early Access Program in Brazil.
Julio Maria Fonseca Chebli, Rogério Serafim Parra, Cristina Flores, Antonio Carlos Moraes, Rodrigo Bremer Nones, Tarcia Nogueira Ferreira Gomes, Ana Maria Bravo Perdomo, Gustavo Scapini, Cyrla Zaltman
Author Information
Julio Maria Fonseca Chebli: Inflammatory Bowel Diseases Center, Federal University of Juiz de Fora, Juiz de Fora 36036-900, Brazil.
Rogério Serafim Parra: Department of Surgery and Anatomy, Ribeirão Preto Medical School, University of São Paulo (USP), Ribeirão Preto 14049-900, Brazil. ORCID
Cristina Flores: Reference Center in Crohn and Colitis, Digestive System Institute, Rio Grande do Sul 90560-002, Brazil. ORCID
Antonio Carlos Moraes: IDOR-Institute for Research and Education, Rio de Janeio 22281-100, Brazil.
Rodrigo Bremer Nones: Gastroenterology Unit, Hospital of Nossa Senhora das Graças, Curitiba 80810-040, Brazil. ORCID
Tarcia Nogueira Ferreira Gomes: Janssen-Cilag Pharmaceutical, São Paulo 04543-011, Brazil.
Ana Maria Bravo Perdomo: Janssen-Cilag Pharmaceutical, São Paulo 04543-011, Brazil. ORCID
Gustavo Scapini: Johnson-Johnson and Internal Former Janssen-Cilag Pharmaceutical, São Paulo 04543-011, Brazil. ORCID
Cyrla Zaltman: Internal Medicine Department, Clementino Fraga Filho University Hospital, Federal University of Rio de Janeiro, Rio de Janeiro 21941-617, Brazil. ORCID
This prospective, observational, open-label study aimed to provide access to ustekinumab prior to market authorization and assess its safety and effectiveness in patients with Crohn's disease (CD) refractory to anti-tumor necrosis factor-α and conventional drugs in Brazil. Patients with a diagnosis of moderate-to-severe active CD for ≥3 months before screening received ustekinumab in a single intravenous induction dose (~6 mg/kg) at week 0, and a 90 mg maintenance dose, subcutaneously, every 8 or 12 weeks, from week 8 through to 80. Serious adverse events (SAE), adverse drug reactions (ADR), clinical response (per CD Activity Index and Harvey Bradshaw Index (HBI) scores), remission (per HBI scores), biomarkers (C-reactive protein (CRP) and fecal calprotectin (FC)) and endoscopic improvement rate over 80 weeks were assessed. Patients with a mean age of 39.9 years were assessed. Discontinuation rate was low (23%) and most adverse events were mild (68.7%). The SAE rate was 21% (mostly infections/infestations or gastrointestinal disorder), and ADR rate was 44%. The CD Activity Index and HBI scores decreased (by 74% and 81%, respectively) with 50% of patients showing normalized CRP and FC, and 63% achieved endoscopic improvement. Ustekinumab was fairly safe, well tolerated and effective in a Brazilian cohort of CD patients.
