Colon to Rectum
Clin Gastroenterol Hepatol. 2024;22(1):154–63.e3
Outcome of biological therapies and small molecules in ulcerative proctitis: A Belgian multicenter cohort study
Background and aims: Several advanced therapies (biologic therapies and small molecules) have been approved for the treatment of moderate-to-severe ulcerative colitis. The registration trials for these agents typically excluded patients with isolated proctitis, leaving an evidence gap. The authors evaluated efficacy and safety of advanced therapies in patients with ulcerative proctitis (UP).
Methods: This multicenter retrospective cohort study included consecutive patients with active UP (Mayo endoscopy subscore ≥ 2, rectal inflammation up to 15 cm) initiating advanced therapy, after failing conventional therapy. The primary end point was short-term steroid-free clinical remission (total Mayo score ≤ 2 with no individual subscore > 1). In addition, drug persistence and relapse-free and colectomy-free survival were assessed. Both binary logistic and Cox regression analyses were performed.
Results: In total, 167 consecutive patients (52.0% female; median age 41.0 years; 82.0% bionaive) underwent 223 courses of therapy for UP (38 adalimumab, 14 golimumab, 54 infliximab, 9 ustekinumab, 99 vedolizumab, 9 tofacitinib). The primary end point was achieved with 36.3% of the treatment courses, and based on multivariate analysis, more commonly attained in bionaive patients (p = 0.001), patients treated with vedolizumab (p = 0.001), patients with moderate endoscopic disease activity (p = 0.002), and a body mass index < 25 kg/m² (p = 0.018). Drug persistence was significantly higher in patients treated with vedolizumab (p < 0.001) and patients with a shorter disease duration (p = 0.006). No new safety signals were observed.
Conclusions: Advanced therapies are also efficacious and safe in patients with ulcerative colitis limited to the rectum. Therefore, the inclusion of patients with ulcerative proctitis in future randomized-controlled trials should be considered.