ABSTRACT Background and Aims While randomised controlled trials have demonstrated the efficacy of ustekinumab for treating Crohn's disease (CD), real‐world effectiveness and patient experience in heterogeneous clinical populations remain less well characterised. We aimed to assess the effectiveness and safety of ustekinumab in routine clinical care incorporating patient‐reported outcomes. Methods This prospective multicentre study recruited adult patients initiating ustekinumab for CD as part of routine clinical care across 22 UK hospitals. Study visits were conducted at baseline and at Weeks 8, 16/20, 56, 80 and 104. Data collected included participant demographics, disease phenotype, previous treatment history, disease activity scores, laboratory measures, dosing regimens, patient‐reported outcomes (patient experience, quality of life QoL and treatment satisfaction) and adverse events. The primary outcome was corticosteroid‐free clinical remission (defined as mHBI < 5) at Week 56. Results Six hundred one participants were recruited between October 2019 and August 2023, with 20% naïve to advanced therapies. At baseline, 43% of participants were assessed as being in clinical remission according to mHBI, whereas only 12% had quiescent disease per the IBD‐Control questionnaire. The primary endpoint was achieved by 33% of participants (95% CI, 30%–37%). Cumulative probabilities of ustekinumab persistence were 78% at 1 year and 63% at 2 years. The IBD‐Control‐8 score showed improvement from baseline to Week 8, increasing from a median of 5 (IQR: 2–10) to 12 (IQR: 8–14), though QoL impairment remained evident in many participants. Most adverse events were mild and consistent with the established safety profile of ustekinumab. Conclusions In this large real‐world cohort, ustekinumab treatment was associated with sustained improvements in both clinical outcomes and QoL measures. Our data suggest that patients treated in clinical practice differ markedly from Phase 3 trial participants. QoL, and not only disease activity as assessed using a conventional disease activity score, may play a significant role in the decision to initiate ustekinumab. Systematic integration of QoL measures into routine care may improve patient care and treatment outcomes.
Young et al. (Thu,) studied this question.