Ustekinumab safety and effectiveness in patients with ulcerative colitis: results from a large real-life study

ABSTRACT

Background

Ustekinumab (UST) is an interleukin-12/interleukin-23 receptor antagonist recently approved for treating ulcerative colitis (UC) but with limited real-world data. Therefore, we evaluated the effectiveness and safety of UST in patients with UC in a real-world setting.

Research design and methods

This is a multicenter, retrospective, observational cohort study. The primary endpoints were the clinical remission rate (partial Mayo score, PMS, ≤1) and the safety of UST. Other endpoints were corticosteroid-free remission (CSFR) rate, clinical response rate (PMS reduction of at least 2 points), and fecal calprotectin (FC) reduction at week 24.

Results

We included 256 consecutive patients with UC (M/F 139/117, median age 52). The clinical remission and clinical response rates at eight weeks were 18.7% (44/235) and 53.2% (125/235), respectively, and 27.6% (42/152) and 61.8% (94/152) at 24 weeks, respectively. At 24 weeks, CSFR was 20.3% (31/152), and FC significantly dropped at week 12 (p = 0.0004) and 24 (p = 0.038). At eight weeks, patients naïve or with one previous biologic treatment showed higher remission (p = 0.002) and clinical >response rates (p = 0.018) than patients previously treated with ≥ 2. Adverse events occurred in six patients (2.3%), whereas four patients (1.6%) underwent colectomy.

Conclusion

This real-world study shows that UST effectively and safely treats patients with UC.

KEYWORDS:

• Real-life
• remission
• safety
• ustekinumab
• ulcerative colitis

Declaration of interests

A Tursi, G Mocci and A Papa have received speaker fees from Janssen. E Savarino has served as a speaker for AbbVie, AGPharma, Alfasigma, Dr Falk, EG Stada Group, Fresenius Kabi, Grifols, Janssen, Innovamedica, Malesci, Pfizer, Reckitt Benckiser, Sandoz, SILA, Sofar, Takeda and Unifarco; has served as a consultant for Alfasigma, Amgen, Biogen, Bristol-Myers Squibb, Celltrion, Diadema Farmaceutici, Dr Falk, Fresenius Kabi, Janssen, Merck & Co, Reckitt Benckiser, Regeneron, Sanofi, Shire, SILA, Sofar, Synformulas GmbH, Takeda and Unifarco; he also received research support from Pfizer, Reckitt Benckiser, SILA, Sofar and Unifarco. G Maconi has served as speaker and/or advisory board fees for AlfaSigma, Arena, Janssen, Gilead and Roche. F Scaldaferri has served as a lecturer for Sanofi. D Pugliese has received speaker fees from AbbVie, MSD, Takeda, Janssen and Pfizer. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

A reviewer on this manuscript has served as speaker, consultant and advisory board of has received research funding from MSD, AbbVie, Janssen, Takeda, Lilly Pharma, Roche, Sandoz, Ferring, Adacyte, Faes Farma, Kern Pharma, Pfizer, Shire Pharmaceuticals, Vifor Pharma, Chiesi and Tillots. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Author contributions

Conception and design: Atursi, G Mocci and A Papa; Acquisition and collection of data: All authors; Analysis and interpretation of data: A Tursi, G Mocci, E Savarino, G Maconi, G Bassotti, W Elisei, M Picchio, AG Gravina, R Pellegrino and A Papa; Drafting of the paper or revising it critically for intellectual content: A Tursi, G Maconi, E Savarino, W Elisei, G Bassotti, M Picchio, AG Gravina, R Pellegrino and A Papa; Final approval of the version to be published: All authors

Ethics statement

The authors state that the study was conducted according to the clinical practice guidelines and following the principles of the Declaration of Helsinki. All patients gave written informed consent before undergoing endoscopy and UST treatment. The reference center (Brotzu Hospital, Cagliari, Italy, PROT. PG/2022/18008) obtained the ethics committee approval for this retrospective study and was accepted by the other centers.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14712598.2024.2309300

Additional information

Funding

This paper was not funded.