Effectiveness of risankizumab induction and maintenance therapy for refractory Crohn’s disease: a real-world experience from a preapproval access programme and early access to medicines scheme

Objective Since approval in Crohn’s disease (CD) of risankizumab, there has been widespread use. Real-world data are, however, limited and our aim is to address that gap. Design/method We performed a retrospective, observational study of risankizumab use in patients with CD starting treatment between January 2021 and January 2023 at two UK centres. Clinical activity, biochemical and faecal biomarkers were measured at baseline, weeks 4, 12, 28 and 52. The primary outcome was clinical response at weeks 12, 28 and 52. Results 53 patients (51% women); median (range) age 40 years (20–70); median disease duration 15 years (6–52). Clinical response was observed in 33% (n=14/42), 45% (n=17/38) and 52% (n=13/25), and clinical remission in 31% (n=13/42), 40% (n=15/38) and 44% (n=11/25) at weeks 12, 28 and 52, respectively. Median C reactive protein decreased from 12 mg/L (IQR: 4–30; n=50) at baseline to 6 mg/L (IQR: 2–16; p=0.03 vs baseline; n=49) at week 12, 3 mg/L (IQR: 2–8, p=0.003; n=44) at week 28 and 3 mg/L (IQR 1–4, p=0.007; n=31) at week 52. Median faecal calprotectin concentration was 668 µg/g (IQR: 246–1098; n=32) at baseline, 298 µg/g (IQR: 176–546, p=NS; n=21) at week 12, 358 µg/g (IQR: 133–622, p=0.03; n=14) at week 28 and 63 µg/g (IQR: 38–120, p=0.007; n=12) at week 52. 12 out of 18 patients discontinued corticosteroids at week 12, 16 by week 28 and 18 by week 52. Four major adverse events—three elective and one emergency surgery—were recorded. Conclusion Risankizumab is effective in a refractory real-world population with CD. Data are available on reasonable request.