Definitions and Outcome Measures in Pediatric Functional Upper Gastrointestinal Tract Disorders: A Systematic Review

Area of Science:

• Pediatric Gastroenterology
• Clinical Trial Methodology
• Functional Gastrointestinal Disorders

Background:

• Functional disorders of the upper gastrointestinal tract are common in children, including functional dyspepsia (FD), cyclic vomiting syndrome (CVS), adolescent rumination syndrome (ARS), and aerophagia.
• Management of these pediatric conditions is challenging, with diverse therapeutic approaches.
• Heterogeneity in definitions and outcome measures across studies complicates treatment evaluation.

Purpose of the Study:

• To systematically assess the definitions and outcome measures used in therapeutic randomized controlled trials (RCTs) for pediatric functional gastrointestinal disorders.
• To identify variations in how FD, CVS, ARS, and aerophagia are defined and their outcomes measured in the literature.

Main Methods:

• A systematic search of CENTRAL, Embase, and MEDLINE/PubMed was conducted up to February 25, 2015.
• Included were English-language therapeutic RCTs or systematic reviews of RCTs involving subjects aged 4–18 years (0–18 for CVS).
• Study quality was assessed using the Delphi list.

Main Results:

• Out of 1398 articles identified, only 8 met the inclusion criteria (7 for FD, 1 for CVS).
• While Rome criteria were generally used for definitions, outcome measures varied significantly across all included studies.
• Seventy-five percent of trials were of good methodological quality, but only 57% utilized validated pain scales.

Conclusions:

• Therapeutic trials for pediatric functional gastrointestinal disorders exhibit considerable variation in outcome measures.
• There is a notable lack of research evaluating treatment regimens for CVS, ARS, and aerophagia.
• Standardized definitions, outcome measures, and validated instruments are essential for enabling meaningful comparisons between intervention studies.