Lubiprostone Found to be Safe, as Effective as Placebo in Children With Constipation

Lubiprostone Found to be Safe, as Effective as Placebo in Children With Constipation 1024 680 Mary Bates, PhD
Close up color photo of little boy holding hands on his belly

The medication, FDA-approved for adults, may benefit older pediatric patients with less of a behavioral component to their constipation.

Pediatric functional constipation, though common, remains challenging to treat. Lubiprostone is a medication approved by the U.S. Food and Drug Administration (FDA) for the treatment of constipation and irritable bowel syndrome with constipation in adults.

In a new study mandated by the FDA, recently published in Clinical Gastroenterology and Hepatology, researchers at Nationwide Children’s Hospital were among the international team evaluating 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.

“Much like every other large, double-blind study 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 only 12 weeks in duration, 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.

The researchers did find that lubiprostone was well tolerated, with a safety profile similar to that in adults.

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, who is also professor of clinical pediatrics at The Ohio State University College of Medicine. “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.

This article is found in the Fall/Winter 2021 print issue. Download the full issue. 


Benninga MA, Hussain SZ, Sood MR, Nurko S, Hyman P, Clifford RA, O’Gorman M, Losch- Beridon T, Mareya S, Lichtlen P, Di Lorenzo C. Lubiprostone for pediatric functional constipation: randomized, controlled, double-blind study with long-term extension. Clinical Gastroenterology and Hepatology. 2021 Apr 7:S1542-3565(21)00393-1.

Image credit: Adobe Stock

About the author

Mary a freelance science writer and blogger based in Boston. Her favorite topics include biology, psychology, neuroscience, ecology, and animal behavior. She has a BA in Biology-Psychology with a minor in English from Skidmore College in Saratoga Springs, NY, and a PhD from Brown University, where she researched bat echolocation and bullfrog chorusing.