Colon to Rectum
Clin Gastroenterol Hepatol. 2022;20(3):602–10.e5
Lubiprostone for pediatric functional constipation: Randomized, controlled, double-blind study with long-term extension
Background and aims: Pediatric functional constipation (PFC) is a common problem in children that causes distress and presents treatment challenges to health care professionals. The authors conducted a randomized, placebo-controlled trial (study 1) in patients with PFC (6–17 years of age) 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 was a phase 3, multicenter, randomized, double-blind, placebo-controlled, 12-week study evaluating the efficacy and safety of lubiprostone 12 μg twice daily (BID) and 24 μg BID. Study 2 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 end point 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 = 0.2245). Both the 12-μg BID and 24-μg 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 pediatric functional constipation but did demonstrate a safety profile similar to that in adults.