Efficacy and safety of mesalamine suppositories for treatment of ulcerative proctitis in children and adolescents

Abstract

Background: Treatment of ulcerative proctitis has not been well studied in pediatric populations. We conducted an open-label trial to evaluate the clinical efficacy of a mesalamine suppository (500 mg) to treat pediatric patients with mild to moderate ulcerative proctitis.

Methods: Pediatric patients (5-17 years of age) with ulcerative proctitis were enrolled for baseline evaluations, including a flexible sigmoidoscopic (or colonoscopic) assessment with biopsies performed at study entry. Eligible patients were started on mesalamine suppositories (500 mg) at bedtime. Two follow-up visits were scheduled after 3 and 6 weeks of treatment. The dose could be increased to 500 mg twice daily at the week 3 follow-up visit if deemed appropriate by the investigator based on the Disease Activity Index (DAI) assessment. The primary outcome measure was a DAI derived from a composite score of stool frequency, urgency of defecation, rectal bleeding, and general well-being.

Results: Forty-nine patients were included in the intent-to-treat analysis. The mean DAI value decreased from 5.5 at baseline to 1.6 and 1.5 at weeks 3 and 6, respectively (P < 0.0001). Only 4 patients had their dose increased to 500 mg twice daily at week 3. Forty-one patients experienced at least one adverse event, most of which were deemed mild and unrelated to study therapy. The most common treatment-emergent adverse events were gastrointestinal (n = 30, 61.2%).

Conclusions: This study showed that a daily bedtime dose of a 500 mg mesalamine suppository is safe and efficacious in children with ulcerative proctitis.

FIGURE 1

Schematic diagram of the study design. HS, once a day at bedtime; BID, twice daily.

FIGURE 2

DAI scores throughout study visits. DAI, Disease Activity Index; ***highly significant from baseline. For week 6 DAI scores the LOCF method was used for missing data.