Effectiveness and Safety of Ustekinumab Maintenance Therapy in 103 Patients With Ulcerative Colitis

A GETAID Cohort Study

Mathurin Fumery; Jérôme Filippi; Vered Abitbol; Amélie Biron; David Laharie; Melanie Serrero; Romain Altwegg; Yoram Bouhnik; Laurent Peyrin-Biroulet; Cyrielle Gilletta; Xavier Roblin; Guillaume Pineton de Chambrun; Lucine Vuitton; Anne Bourrier; Stephane Nancey; Jean-Marc Gornet; Stephane Nahon; Guillaume Bouguen; Stephanie Viennot; Maria Nachury; Aurelien Amiot


Aliment Pharmacol Ther. 2021;54(7):944-951. 

In This Article

Abstract and Introduction


Background: Phase III trials have demonstrated the efficacy and safety of ustekinumab in ulcerative colitis (UC), but few real-life long-term data are currently available.

Aims: To assess the real-world effectiveness and safety of ustekinumab in patients with UC.

Methods: From January to September 2019, all consecutive patients with active UC treated with ustekinumab in a GETAID centre were included. Patients were evaluated at week 52. Remission was defined as a partial Mayo Clinic score ≤2.

Results: We included 103 patients with UC (62 men; mean age: 41.2 ± 16.2 years; 52% pancolitis E3) with an insufficient response to immunosuppressants, anti-TNFs and/or vedolizumab. At week 52, 45 (44%) patients had discontinued ustekinumab mainly due to lack of effectiveness (n = 41). The cumulative probabilities of ustekinumab persistence were 96.1%, 81.6%, 71.7% and 58.4% after 3, 6, 9 and 12 months respectively. The overall steroid-free clinical remission rate at week 52 was 32% of whom 71% had subscores of null for rectal bleeding and stool frequency. Ten patients underwent colectomy within a median of 6.7 [4.3–10.6] months. Adverse effects were observed in 15 (16.9%) patients; 4 (4.5%) were severe, including one patient who died from a myocardial infarction.

Conclusion: After 52 weeks, over one-half of patients with refractory UC were still treated by ustekinumab and one-third were in steroid-free clinical remission.


Biologics, such as anti-TNFs and vedolizumab have dramatically changed the management of ulcerative colitis (UC).[1–4] However, these current therapies are limited by a lack of effectiveness, as only one-third of treated patients achieves long-term remission.[5–7]

Ustekinumab (Janssen Biotech Inc), a fully human IgG1 monoclonal antibody targeting the IL-12/IL-23 shared p40 subunit, was approved for the treatment of psoriasis, psoriatic arthritis and Crohn's disease. In a phase III trial for the treatment of moderate-to-severe UC, ustekinumab induced a response at 8 weeks and maintained clinical benefit through 52 weeks of treatment in patients who had an inadequate response or unacceptable side effects from corticosteroids, immunomodulators, anti-TNF agents and/or vedolizumab.[8] Based on these results, ustekinumab has been approved by the European Medicines Agency for the treatment of patients with moderate-to-severe UC and reimbursed in France in patients with failure or contra-indication to at least one biologic.

Given the lack of real-world data, we recently reported short-term effectiveness and safety of ustekinumab in a multicentre retrospective cohort of 103 patients with refractory UC.[9] At weeks 12–16, steroid-free clinical remission and clinical remission rates were observed in 35% and 40% of patients with a good safety profile respectively. However, long-term real-world data on ustekinumab in UC are lacking.

The aims of this study were to evaluate for the first time the long effectiveness and safety of ustekinumab maintenance therapy in a multicentre cohort study from the GETAID and to identify associated factors of ustekinumab failure.