Outcome of Biological Therapies and Small Molecules in Ulcerative Proctitis: A Belgian Multicenter Cohort Study
Pauline Lemmens1, Edouard Louis2, Wouter Van Moerkercke3
1Department of Gastroenterology and Hepatology, University Hospitals Leuven, Leuven, Belgium.
Background & 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. We 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 of ≥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 = .001), patients treated with vedolizumab (P = .001), patients with moderate endoscopic disease activity (P = .002), and a body mass index <25 kg/m2 (P = .018). Drug persistence was significantly higher in patients treated with vedolizumab (P < .001) and patients with a shorter disease duration (P = .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 UP in future randomized-controlled trials should be considered.
Insights
Advanced therapies show efficacy and safety for ulcerative proctitis (UP), a condition often excluded from trials. Vedolizumab demonstrated superior drug persistence in this patient group.
Area of Science:
- Gastroenterology and Hepatology
- Clinical Pharmacology
- Inflammatory Bowel Disease Research
Background:
- Advanced therapies (biologics, small molecules) are approved for moderate-to-severe ulcerative colitis.
- Clinical trials often exclude patients with isolated proctitis, creating an evidence gap for this specific population.
- Ulcerative proctitis (UP) is characterized by rectal inflammation up to 15 cm.
Purpose of the Study:
- To evaluate the efficacy and safety of advanced therapies in patients diagnosed with ulcerative proctitis (UP).
- To assess short-term steroid-free clinical remission, drug persistence, and survival outcomes (relapse-free, colectomy-free) in UP patients.
- To identify factors influencing treatment success in UP patients receiving advanced therapies.
Main Methods:
- Multicenter retrospective cohort study of 167 consecutive patients with active UP.
- Patients initiated advanced therapy after failing conventional treatments.
- Primary endpoint: steroid-free clinical remission. Secondary endpoints: drug persistence, relapse-free survival, colectomy-free survival. Statistical analyses included logistic and Cox regression.
Main Results:
- 36.3% of treatment courses achieved short-term steroid-free clinical remission.
- Remission was more common in bio-naive patients, those treated with vedolizumab, patients with moderate endoscopic activity, and lower BMI (<25 kg/m²).
- Vedolizumab and shorter disease duration were associated with significantly higher drug persistence. No new safety signals were identified.
Conclusions:
- Advanced therapies are effective and safe for treating ulcerative proctitis (rectal-limited ulcerative colitis).
- Consideration should be given to including UP patients in future randomized-controlled trials for advanced therapies.
- Vedolizumab shows promise for improved drug persistence in UP patients.
Related Concept Videos
01:29Drugs for Treatment of Ulcerative Colitis in IBD
01:29Inflammatory Bowel Disease IV: Pharmacological Management
Pharmacologic...
01:20Drugs for Peptic Ulcer Disease: Prostaglandin Analogs as Mucosal Protective Agents
Non-steroidal anti-inflammatory drugs (NSAIDs) can induce peptic ulcers by inhibiting cyclooxygenase, decreasing...
01:16Treating Helicobacter pylori in Peptic Ulcers: Antimicrobial Therapy
01:17Drugs for Treatment of Diarrhea-Predominant IBS
Two specific drugs used in the treatment are alosetron (Lotronex) and eluxadoline (Viberzi). Alosetron, a 5-HT3 antagonist, works by slowing the movement of stools in the gut, reducing bowel...
01:30Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy
Diagnostic studies
A colonoscopy is the definitive screening test, distinguishing ulcerative colitis from other colon diseases with similar symptoms. During a colonoscopy test, inflamed mucosa with exudate ulcerations can be observed, and biopsies are taken to determine the histologic characteristics of the...