Budesonide foam versus budesonide enema in active ulcerative proctitis and proctosigmoiditis

Background Rectal budesonide is an effective treatment of active ulcerative proctitis or proctosigmoiditis. Aim To compare the therapeutic efficacy, tolerability and safety, and patient's preference of budesonide foam vs. budesonide enema. Methods Patients with active ulcerative proctitis or proctosigmoiditis (clinical activity index > 4 and endoscopic index >= 4) were eligible for this double-blind, double-dummy, randomized, multicentre study. They received 2 mg/25 mL budesonide foam and placebo enema (n = 265), or 2 mg/100 mL budesonide enema and placebo foam (n = 268) for 4 weeks. Primary endpoint was clinical remission (clinical activity index <= 4) at the final/withdrawal visit (per protocol). Results A total of 541 patients were randomized - 533 were evaluable for intention-to-treat analysis and 449 for per protocol analysis. Clinical remission rates (per protocol) were 60% for budesonide foam and 66% for budesonide enema (P = 0.02362 for non-inferiority of foam vs. enema within a predefined non-inferiority margin of 15%). Both formulations were safe and no drug-related serious adverse events were observed. Because of better tolerability and easier application most patients preferred foam (84%). Conclusion Budesonide foam is as effective as budesonide enema in the treatment of active ulcerative proctitis or proctosigmoiditis. Both budesonide formulations are safe, and most patients prefer foam.