Related Experiment Video
Updated: Aug 12, 2026
Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
Published on: February 11, 2017
Placebo-controlled trial of misoprostol in cystic fibrosis
1Department of Thoracic Medicine, Royal Children's Hospital, Melbourne, Australia.
Insights
Misoprostol improved fat absorption in children with cystic fibrosis who had less than 90% absorption on pancreatic enzyme therapy alone. It did not significantly benefit those with over 90% absorption.
Area of Science:
- Pediatric Gastroenterology
- Pharmacology
Background:
- Cystic fibrosis (CF) often leads to malabsorption due to pancreatic insufficiency.
- Pancreatic enzyme replacement therapy (PERT) is standard, but some patients experience residual malabsorption.
Purpose of the Study:
- To evaluate the efficacy of misoprostol in improving fat absorption in pediatric CF patients on PERT.
- To identify patient subgroups that may benefit from adjunctive misoprostol therapy.
Main Methods:
- A 6-week, double-blind, placebo-controlled, crossover trial involving 17 children with CF.
- Patients received either misoprostol (100 mcg, 4 times daily) or placebo, followed by the alternative treatment.
- Fat absorption was measured during both treatment periods.
Main Results:
- Misoprostol significantly improved fat absorption in patients with baseline absorption <90% (p<0.01).
- No significant improvement was observed in patients with baseline absorption >90%.
- One patient showed elevated eosinophils; other hematological and biochemical parameters remained unchanged.
Conclusions:
- Misoprostol is beneficial for children with cystic fibrosis experiencing residual fat malabsorption despite standard enzyme therapy.
- It may be a valuable adjunct for specific CF patient subgroups with suboptimal absorption.
- Further research into safety and optimal dosing in CF is warranted.
Abstract:
Seventeen children with cystic fibrosis were studied in a 6-week double-blind, placebo-controlled, crossover trial to determine the efficacy of misoprostol, 100 micrograms, four times a day, in improving fat absorption in patients already on pancreatic enzyme therapy. In those patients who had greater than 90% absorption on enzyme therapy alone, no further significant increase in absorption was achieved with misoprostol administration. Those patients who had absorption of less than 90% on standard enzyme therapy showed a significant improvement with misoprostol administration (p less than 0.01). One patient had a significant elevation in the eosinophil count during the period of misoprostol administration, but there were no significant changes in any other hematological or biochemical parameter. Misoprostol appears to be of benefit to those children with cystic fibrosis who have residual malabsorption on standard enzyme therapy.
Related Concept Videos
01:23Cystic Fibrosis: Pathogenesis
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation, but...
01:20Drugs for Peptic Ulcer Disease: Prostaglandin Analogs as Mucosal Protective Agents
Non-steroidal anti-inflammatory drugs (NSAIDs) can induce peptic ulcers by inhibiting cyclooxygenase, decreasing...
01:21Drugs for Treatment of Constipation-Predominant IBS