Editorial: Effectiveness and Safety of Ustekinumab in Ulcerative Colitis—Where We Are Now in Real Life. Authors' Reply

We appreciate the interest shown by Dr. Mocci and Tursi in our study, and their acknowledgment of its importance in advancing the understanding of the role of ustekinumab in the management of ulcerative colitis (UC) in clinical practice [1]. Real-world studies provide necessary and complementary information to clinical trials regarding the benefits of drugs in clinical practice. First, only one-third of individuals with inflammatory bowel disease receiving a treatment in clinical practice would have been eligible to participate in clinical trials [2]. Second, the definition of disease activity differs in clinical trials, which biases inclusion towards a certain type of patient. Third, clinical trials do not always provide the ability to optimise the dose. Finally, the endpoints of interest may differ.

As noted by Mocci and Tursi our study included a large number of patients (620) with UC treated with ustekinumab in clinical practice, with relatively long follow-up (median 12 months) [3]. Our results support the effectiveness and safety profile of ustekinumab in a cohort of patients with refractory UC. Additionally, this study allowed us to identify factors associated with loss of response and drug discontinuation. Finally, we described that empiric dose escalation restores the drug's efficacy in over 60% of patients, making it a recommended strategy in this clinical scenario.

Furthermore, Mocci and Tursi highlighted some limitations of our study [1]. As acknowledged in the manuscript, the study was retrospective. However, our main outcome was treatment survival and these data were readily available in patients' medical records, making recall bias unlikely. However, while the definition of clinical remission is not standardised, most clinical practice studies and clinical trials on drugs for UC have similarly used a Partial Mayo Score ≤ 2 [4, 5]. Finally, we would like to highlight that patients discontinuing ustekinumab for any reason before their last visit were considered treatment failures, reflecting the rigour of our evaluation.

In conclusion, thanks to this large cohort of patients with UC, we were able to describe in detail the persistence, effectiveness and safety of ustekinumab in a real-world setting. We appreciate the opportunity to share the results of our study and hope they will be useful for decision-making in clinical practice. We encourage clinicians to share their real-world experience, as these data are essential for the proper positioning of the drug in clinical practice.

María Chaparro: conceptualization, writing – review and editing, writing – original draft. Javier P. Gisbert: conceptualization, writing – review and editing.

María Chaparro has served as speaker, consultant or received research or education funding from MSD, Abbvie, Hospira, Pfizer, Takeda, Janssen, Ferring, Shire Pharmaceuticals, Dr. Falk Pharma, Tillotts Pharma, Biogen, Gilead and Lilly. Javier P. Gisbert has served as speaker, consultant and advisory member for or has received research funding from MSD, Abbvie, Pfizer, Kern Pharma, Biogen, Mylan, Takeda, Janssen, Roche, Sandoz, Celgene/Bristol Myers, Gilead/Galapagos, Lilly, Ferring, Faes Farma, Shire Pharmaceuticals, Dr. Falk Pharma, Tillotts Pharma, Chiesi, Casen Fleet, Gebro Pharma, Otsuka Pharmaceutical, Norgine and Vifor Pharma.

This article is linked to Chaparro et al papers. To view these articles, visit https://doi.org/10.1111/apt.18230 and https://doi.org/10.1111/apt.18272.