Safety of Lubiprostone in Pediatric Patients With Functional Constipation: A Nonrandomized, Open-Label Trial

Area of Science:

• Gastroenterology
• Pediatric Medicine
• Clinical Pharmacology

Background:

• Pediatric functional constipation (PFC) affects a significant portion of children, with current treatments offering limited sustained relief.
• Lubiprostone, a chloride channel activator, enhances intestinal fluid secretion and motility.
• Its safety and efficacy in pediatric populations require thorough investigation.

Purpose of the Study:

• To evaluate the safety and tolerability of oral lubiprostone in children diagnosed with pediatric functional constipation (PFC).
• To assess the efficacy of lubiprostone in managing PFC symptoms over a 24-week treatment period.

Main Methods:

• A phase 3, open-label safety trial involving 87 pediatric patients (ages 6-17 years) with PFC.
• Patients received either 12 mcg or 24 mcg of lubiprostone twice daily based on weight (<50 kg and ≥50 kg, respectively) for 24 weeks.
• Safety endpoints included treatment-emergent adverse events (TEAEs), laboratory parameters, and vital signs; efficacy was assessed via investigator ratings.

Main Results:

• Lubiprostone demonstrated good tolerability in the pediatric population, with TEAEs comparable to previous trials.
• Gastrointestinal disorders like diarrhea and vomiting were the most frequent mild TEAEs; upper abdominal pain was the only TEAE leading to discontinuation in more than one patient.
• Investigator assessments indicated treatment effectiveness, with mean ratings of 2.7-2.9 at week 12 and 2.2-2.7 at week 24 for the 12-mcg and 24-mcg groups, respectively.

Conclusions:

• Oral lubiprostone is well-tolerated and shows promise for treating pediatric functional constipation (PFC).
• The safety profile in children is consistent with findings in adult trials, supporting its use in this age group.