Protocol for a Randomized Controlled Trial to Determine if Biomarkers Predict Response to a Pediatric Chronic Pain...

Area of Science:

• Pediatric gastroenterology
• Neurogastroenterology
• Biomedical research

Background:

• Disorders of Gut-Brain Interaction (DGBI) affect 15-20% of children and adults, causing chronic abdominal pain and disability.
• Current management strategies like cognitive behavioral therapy (CBT) and the low FODMAP diet (LFD) show limited efficacy, improving symptoms in only ~40% of patients.
• Biomarkers and gut microbiome composition may influence treatment response, but this has not been studied in pediatric DGBI trials.

Purpose of the Study:

• To identify physiological characteristics that predict differential treatment response to CBT versus LFD in children with DGBI.
• To compare the effectiveness of CBT and LFD in children with and without specific abnormal physiological biomarkers.
• To optimize management strategies for DGBI by matching interventions to individual patient profiles.

Main Methods:

• A randomized controlled trial involving 200 children aged 7-12 years with DGBI.
• Categorization of participants based on Autonomic Nervous System imbalance and/or gut physiology abnormalities.
• Randomization to either CBT or LFD to assess treatment effectiveness in relation to identified physiological biomarkers.

Main Results:

• The study hypothesizes that CBT will be more effective in children without abnormal physiology, while LFD will be more effective in those with abnormal physiology.
• Primary outcomes include improvements in abdominal pain frequency/severity and health-related quality of life.
• This study is the first to link physiological biomarkers to treatment response in pediatric DGBI.

Conclusions:

• This research pioneers the identification of physiological moderators for DGBI treatment.
• Findings may enable personalized medicine approaches, improving outcomes for children with chronic abdominal pain.
• Timely application of the most effective intervention based on individual characteristics can reduce the burden of DGBI.