Real-world data on clinical response to inflammatory bowel disease biological treatments in patients with concurrent primary sclerosing cholangitis: a case-control study

BACKGROUND: The intestinal therapy response in patients with primary sclerosing cholangitis-associated inflammatory bowel disease (PSC-IBD) is not well explored. This study compared the intestinal therapy response to biological therapies in patients with PSC and IBD (PSC-IBD group) vs patients with IBD alone (IBD group). METHODS: We performed a case–control study and identified 46 patients in the PSC-IBD group and 180 in the IBD group who were treated with infliximab, vedolizumab, and/or ustekinumab at the IBD outpatient clinic of Charité-Universitätsmedizin Berlin, Campus-Virchow-Klinikum. To account for differences in demographics and disease characteristics, propensity score matching (ratio 1:1) was performed. The primary outcome was clinical therapy response within 20 weeks, defined as remission (partial Mayo Score ≤ 1 or Harvey-Bradshaw-Index < 5) or partial response (decrease of partial Mayo Score ≥ 2 or a decrease of Harvey-Bradshaw-Index > 3). Secondary outcomes included treatment persistence, endoscopic response, decrease in faecal calprotectin, concomitant medication and safety. RESULTS: After matching, 46 patients per group were analysed (median follow-up: PSC-IBD: 44 months; IBD: 60 months), with a total of 136 treatments administered. The cohort demographics and disease characteristics were balanced between both groups. While clinical response rates did not significantly differ between groups in patients receiving infliximab or vedolizumab, in patients receiving ustekinumab, clinical response rates within the first 20 weeks were significantly lower in the PSC-IBD group (9 of 21, 42.9%) compared with the IBD group (20 of 26, 76.9%; P = 0.017). Treatment persistence after 12 months of patients on biological therapy did not differ significantly between groups across all therapies, but a numerically lower persistence rate was observed for ustekinumab in the PSC-IBD group (6 of 21, 28.6%) compared with the IBD group (10 of 22, 45.5%; P = 0.252). CONCLUSION: Infliximab and vedolizumab appear equally effective for achieving clinical response in patients with PSC-IBD and IBD, while the early clinical response to ustekinumab was significantly lower in patients with PSC-IBD.