Clin Gastroenterol Hepatol. 2021 Apr 7:S1542-3565(21)00393-1. doi: 10.1016/j.cgh.2021.04.005. Online ahead of print.
ABSTRACT
BACKGROUND & AIMS: Pediatric functional constipation (PFC) is a common problem in children that causes distress and presents treatment challenges to healthcare professionals. We conducted a randomized, placebo-controlled trial (Study 1) in patients with PFC (aged 6-17 years) to evaluate the efficacy and safety of lubiprostone, followed by an open-label extension for those who completed the placebo-controlled phase (Study 2).
METHODS: Study 1 (NCT02042183) was a phase 3, multicenter, randomized, double-blind, placebo-controlled, 12-week study evaluating the efficacy and safety of lubiprostone 12 mcg twice-daily (BID) and 24 mcg BID. Study 2 (NCT02138136) was a phase 3, long-term, open-label extension of Study 1. In both studies, lubiprostone doses were based on patients’ weight. Efficacy was assessed solely based on Study 1, with a primary endpoint of overall spontaneous bowel movement (SBM) response (increase of ≥1 SBM/week vs baseline and ≥3 SBMs/week for ≥9 weeks, including 3 of the final 4 weeks).
RESULTS: 606 patients were randomized to treatment (placebo: n=202; lubiprostone: n=404) in Study 1. No statistically significant difference in overall SBM response rate was observed between the lubiprostone and placebo groups (18.5% vs 14.4%; P=.2245). Both the 12-mcg BID and 24-mcg BID doses of lubiprostone were well tolerated in the double-blind and extension phases, with a safety profile consistent with that seen in adult studies.
CONCLUSIONS: Lubiprostone did not demonstrate statistically significant effectiveness over placebo in children and adolescents with PFC, but did demonstrate a safety profile similar to that in adults.
PMID:33838349 | DOI:10.1016/j.cgh.2021.04.005
