Development of a core outcome set for clinical trials in childhood constipation: a study using a Delphi technique

Area of Science:

• Pediatric Gastroenterology
• Clinical Trial Methodology
• Outcome Research

Background:

• Functional constipation (FC) in children presents varied symptoms, leading to differing perceptions among patients, parents, and healthcare professionals (HCPs).
• This discrepancy results in a lack of consensus on definitions and outcomes used in FC therapeutic trials.
• Standardized outcome measures are crucial for reliable and comparable research in pediatric FC.

Purpose of the Study:

• To develop a consensus-based core outcome set (COS) for functional constipation (FC) in children aged 0-18 years.
• To establish a standardized set of outcomes for use in clinical trials involving pediatric FC.
• To improve the consistency and comparability of research in childhood FC.

Main Methods:

• A Delphi technique was employed, involving healthcare professionals (HCPs), parents of children with FC, and patients aged 12-18 years.
• Participants identified and prioritized relevant outcomes through multiple survey phases.
• A final COS was determined through a face-to-face expert meeting.

Main Results:

• The study involved 109 HCPs, 165 parents, and 50 children in the initial phase, with high response rates.
• The final core outcome set (COS) for pediatric functional constipation includes: defecation frequency, stool consistency, painful defecation, quality of life, treatment side effects, fecal incontinence, abdominal pain, and school attendance.
• This COS is applicable to children of all ages (0-18 years).

Conclusions:

• The developed COS for pediatric functional constipation (FC) will reduce heterogeneity in clinical trials.
• Implementing this COS will enhance the comparability of studies investigating FC treatments in children.
• Researchers are strongly encouraged to adopt this COS in future therapeutic trials for childhood FC.