Do Most Children with Functional Constipation Meet the Commonly Used Clinical Trial Endpoints?

Area of Science:

• Pediatric Gastroenterology
• Gut-Brain Interaction Disorders
• Clinical Trial Methodology

Background:

• Functional constipation (FC) diagnosis relies on Rome IV criteria, requiring at least two of seven symptoms.
• Common clinical trial endpoints for FC include bowel movement frequency, stool consistency, and fecal incontinence.
• Limited data exists on how well these endpoints represent symptom distribution in children with FC.

Purpose of the Study:

• To assess the frequency of each Rome IV criterion in a large, community-based sample of children.
• To determine if commonly used clinical trial endpoints accurately reflect the symptom distribution in pediatric FC.
• To identify potential disparities between clinical trial practices and real-world symptom prevalence.

Main Methods:

• Cross-sectional study involving 6611 school children aged 8-18 years across seven Colombian cities.
• Participants completed the Pediatric Gastrointestinal Symptoms Rome IV Questionnaire (QPGS-IV).
• Analysis included the prevalence of FC and the distribution and combination of diagnostic criteria.

Main Results:

• Functional constipation (FC) was diagnosed in 12.8% of children, the most common gut-brain interaction disorder.
• The most prevalent Rome IV criteria were infrequent stools (<2/week, 66.1%) and painful bowel movements (65%).
• Fecal incontinence was infrequent (6.9%), and 60.5% met only two criteria, commonly infrequent stools and painful bowel movements.

Conclusions:

• Significant variability exists in Rome IV criteria prevalence for pediatric FC in a community setting.
• Painful bowel movements are a critical diagnostic component but are underutilized as a clinical trial endpoint.
• Reassessing endpoint selection in pediatric FC clinical trials is suggested to better align with real-world symptom distribution.