Objective Ustekinumab is an interleukin-12/interleukin-23 receptor antagonist licensed for the treatment of ulcerative colitis (UC). Clinical trial data were promising; however, real-world data are limited. We assessed the safety and effectiveness of ustekinumab in UC in a real-world setting.
Design/method This was a multicentre, retrospective, observational cohort study between February 2020 and January 2022. Disease activity was assessed using the Simple Clinical Colitis Activity Index (SCCAI). Clinical remission was defined as a SCCAI≤2. The primary endpoints were rates of corticosteroid-free remission (CSFR) at week 16 and at week 26. Objective outcomes, including faecal calprotectin (FCAL), were also collected.
Results 110 patients with UC (65% male; median age 40 (IQR range 29–59); 96% with prior biologic and/or tofacitinib exposure) had a median follow-up of 28 weeks (IQR 17–47). CSFR was 36% (18/50) at week 16% and 33% (13/39) at week 26, corresponding with a significant fall in SCCAI from 6 (IQR 4–8) at baseline to 3 (IQR 0–5) at week 26, p<0.001. By week 16, there was improvement of median FCAL measurements, which fell from a baseline of 610 µg/g (IQR 333–1100) to 102 µg/g (IQR 54–674) at week 16. At the end of follow-up, 15% (17/110) had discontinued treatment; 13 patients due to primary non-response or loss of response, and 1 patient for family planning. Treatment was discontinued in three patients due to adverse events.
Conclusion In the largest real-world study to date, ustekinumab was effective with a reassuring safety profile in a refractory cohort of patients.
- ULCERATIVE COLITIS
- IBD CLINICAL
Data availability statement
No data are available. Individual patient data cannot be shared for confidentiality reasons.
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Contributors SH and MAS: conception and study design. SH, LA-H, SB, AB, RL and LM data collection. SH data analysis, data interpretation, writing the manuscript and guarantor. All authors critically reviewed the manuscript before submission.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests SH has served as a speaker, a consultant, and/or advisory board member for Pfizer, Janssen, Abbvie and Takeda, with research supported by Pfizer and Galápagos NV. AB has received speaker fees from Takeda and meeting support fees from Abbvie, Dr Falk and Vifor Pharma. KBK served as a speaker, a consultant, and/or advisory board member for Janssen, Takeda, PredictImmune, Galapagos, Ferring, and Amgen. KP has received honoraria for educational meetings and speaker fees from Abbvie, Janssen, Takeda, DrFalk, PredictImmune and Ferring and has received advisory board fees from Abbvie, Galapagos and Janssen. PMI has received lecture fees from Abbvie, Warner Chilcott, Ferring, Falk Pharma, Takeda, MSD, Johnson and Johnson, Shire, and Pfizer, financial support for research from MSD, Takeda, and Pfizer, advisory fees from Abbvie, Warner Chilcott, Takeda, MSD, Vifor Pharma, Pharmacosmos, Topivert, Genentech, Hospira, and Samsung Bioepis. MAS served as a speaker, a consultant, and/or an advisory board member for Sandoz, Janssen, Takeda, MSD, Falk, Abbvie, Bristol Myers Squibb, Galapagos, and Samsung Bioepis. LA-H, SB, LM, RL and AW report no conflicts of interest.
Provenance and peer review Not commissioned; externally peer reviewed.
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