Pediatric functional constipation remains challenging to treat. Lubiprostone, a medication approved by the U.S. Food and Drug Administration (FDA), treats constipation and irritable bowel syndrome with constipation in adults.
A new FDA mandated study, published in Clinical Gastroenterology and Hepatology, evaluated the efficacy and safety of lubiprostone in children with constipation.
The researchers conducted a double-blind, randomized, placebo-controlled study and a long-term, open-label extension study in over 600 children 6-17 years of age with pediatric functional constipation. The results showed that while well tolerated and safe, the efficacy was the same as a placebo.
“Similarly to other double-blind studies on pediatric constipation, we found lubiprostone was as effective as placebo in treating children with chronic constipation,” says Carlo Di Lorenzo, MD, chief of the Division of Pediatric Gastroenterology, Hepatology and Nutrition at Nationwide Children’s and senior author of the study.
Dr. Di Lorenzo says he was not surprised that the medication did not show the same efficacy in children as in adults. “Pediatric constipation is usually a behavioral issue, a completely different condition from constipation in adults,” he says.
Dr. Di Lorenzo points to a few factors affecting their results. The first part of the study was 12 weeks long, often not enough time for children who withhold their stool because they are afraid it will hurt to overcome their fear. In addition, one of the outcome measures mandated by the FDA — frequency of bowel movements — may not be as relevant in children. While this measure is appropriate for adults with constipation, Dr. Di Lorenzo says that children with constipation complain more about hard stool or fecal incontinence than infrequent bowel movements.
According to Dr. Di Lorenzo, the fact that lubiprostone was as effective as placebo does not mean it was ineffective.
“In the right patients, this medication worked well,” says Dr. Di Lorenzo. “It was probably not beneficial in patients that withhold their stool. If a child is going to withhold their stool, there is no medication that will help until they overcome their fear.”
Dr. Di Lorenzo suggests that lubiprostone might be more appropriate for adolescent patients experiencing constipation with no behavioral issues.
Based on these results, the FDA-approved labeling for lubiprostone has been updated to state that its safety and effectiveness have not been established in pediatric patients under 6 years of age, and that effectiveness has not been established in pediatric patients 6 years and older.