title: Recommendations for pharmacological clinical trials in children with irritable bowel syndrome: the Rome foundation pediatric subcommittee on clinical trials.
abstract: BACKGROUND
  There is little published evidence of efficacy for the most commonly used treatments. Thus, there is an urgent need to conduct clinical trials on existing and novel therapies.
  
  
  OBJECTIVE
  In order to address these issues the Rome Foundation and members of the Pediatric Committee of the European Medicines Agency formed a subcommittee on clinical trials to develop guidelines for the design of clinical trials in children with irritable bowel syndrome (IBS). The following recommendations are based on evidence from published data when available and expert opinion.
  
  
  CONCLUSIONS
  The subcommittee recommends randomized, double-blind, placebo-controlled, parallel-group, clinical trials to assess the efficacy of new drugs. The combined endpoints for abdominal pain are a decrease in intensity of at least 30% compared with baseline and to meet or exceed the Reliable Change Index (RCI) for the sample. Stool consistency is measured with the Bristol Stool Scale Form (BSFS). The subcommittee recommends as entry criteria for abdominal pain a weekly average of worst abdominal pain in past 24 h of at least 3.0 on a 0-10 point scale or at least 30 mm in 100 mm Visual Analog Scale. For stool endpoints the committee recommends an average stool consistency lower than 3 in the BSFS during the run-in period for clinical trials on IBS-C and an average stool consistency greater than 5 in the BSFS during the run-in period for clinical trials on IBS-D. Changes in stool consistency are the primary endpoints for both IBS with diarrhea (IBS-D) and IBS with constipation (IBS-C).
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology & Nutrition, Nationwide Children's Hospital, Columbus, OH, USA. miguel.saps@nationwidechildrens.org.
authors.forename: M
authors.initials: M
authors.lastname: Saps
authors.affiliation: Division of Gastroenterology and Hepatology, Center for Functional Gastrointestinal and Motility Disorders, University of North Carolina, Chapel Hill, NC, USA.
authors.forename: M A L
authors.initials: MA
authors.lastname: van Tilburg
authors.affiliation: Department of Child and Adolescent Psychiatry, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.|Feinberg School of Medicine, Northwestern University, Chicago, IL, USA.|Mary Ann and J. Milburn Smith Child Health Research Program, Chicago, IL, USA.|Children's Hospital of Chicago Research Center, Chicago, IL, USA.
authors.forename: J V
authors.initials: JV
authors.lastname: Lavigne
authors.affiliation: Division of Pediatric Gastroenterology Hepatology & Nutrition, Medical College of Wisconsin, Milwaukee, WI, USA.
authors.forename: A
authors.initials: A
authors.lastname: Miranda
authors.affiliation: Department of Pediatric Gastroenterology and Nutrition, Emma Children's Hospital/Academic Medical Center, Amsterdam, The Netherlands.
authors.forename: M A
authors.initials: MA
authors.lastname: Benninga
authors.affiliation: Member of the Pediatric Committee (PDCO) European Medicines Agency, London, UK.
authors.forename: J A
authors.initials: JA
authors.lastname: Taminiau
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology & Nutrition, Nationwide Children's Hospital, Columbus, OH, USA.
authors.forename: C
authors.initials: C
authors.lastname: Di Lorenzo
mesh_terms: D015746:Abdominal Pain / Q000175:diagnosis / Q000188:drug therapy / Q000453:epidemiology; D002648:Child; D004311:Double-Blind Method; D005582:Foundations / Q000592:standards*; D005765:Gastrointestinal Agents / Q000627:therapeutic use; D006801:Humans; D043183:Irritable Bowel Syndrome / Q000175:diagnosis / Q000188:drug therapy* / Q000453:epidemiology; D018579:Patient Selection; D017410:Practice Guidelines as Topic / Q000592:standards*; D016032:Randomized Controlled Trials as Topic / Q000379:methods / Q000592:standards*; D012384:Rome
chemical_list: D005765:Gastrointestinal Agents
keywords: abdominal pain; children; clinical trials; endpoints; irritable bowel syndrome; stool consistency