Symptom measurement in pediatric chronic idiopathic constipation (CIC) and irritable bowel syndrome with constipation (IBS-C) trials requires appropriately developed clinical outcome assessments (COAs).
Literature was reviewed to identify symptom COAs meeting regulatory standards. Searches were conducted in Pubmed/Medline, EMBASE, and PsychINFO. Title/abstracts were reviewed to identify qualitative studies and those using COAs to measure pediatric CIC/IBS symptoms. Pediatric functional gastrointestinal experts provided input on relevant symptom-concepts to measure.
Review of 1,105 abstracts identified 1 relevant qualitative article and 113 articles including COAs. Symptoms most frequently measured in CIC studies were frequency of bowel movements, fecal incontinence/encopresis, abdominal pain, stool consistency, and painful defecation. Symptoms most frequently measured in IBS were abdominal pain, abdominal distention/bloating, stool consistency, frequency of bowel movements, and gas. Evidence of development/validity of COAs was limited. Expert feedback was broadly consistent with the literature.
Findings demonstrate consistency in the literature on key CIC/IBS symptoms to measure in pediatric trials, but existing COAs do not meet regulatory standards.
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The authors thank the following expert physicians who participated and shared their views in the Expert Advisory Meeting reported in this article: Warren P. Bishop, Carlo Di Lorenzo, Jeffrey S. Hyams, Paul Hyman, Anthony Lembo, Adrian Miranda, and Miguel Saps.
Disclosure of financial arrangements
This research was funded by Forest Research Institute and Ironwood Pharmaceuticals.
Conflict of interest
Robert Arbuckle, Linda Abetz-Webb, and Elizabeth Gargon are employees of Adelphi Values (or were employees at the time the research was conducted), a health outcomes agency contracted by Forest Research Institute and Ironwood Pharmaceuticals to perform the research. Robyn Carson and Steven Shiff are employees of Forest Research Institute. Barbara Lewis, Caroline Kurtz, and Jeffrey Johnston are employees of Ironwood Pharmaceuticals (or were employees at the time the research was conducted). Jeffrey Hyams and Carlo Di Lorenzo were independent consultants contracted by Forest Research Institute and Ironwood Pharmaceuticals for their participation in the research.
Robert Arbuckle, Linda Abetz-Webb, and Elizabeth Gargon contributed to the study design, performed the literature review, and prepared the first draft of the manuscript. Robyn Carson, Jeffrey Hyams, Carlo Di Lorenzo, Barbara Lewis, Caroline Kurtz, Steven Shiff, and Jeffrey Johnston contributed to the design of the study, provided strategic input into the literature review, and reviewed and approved the manuscript. Rob Arbuckle acts as the overall guarantor for this manuscript.
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Arbuckle, R.A., Carson, R.T., Abetz-Webb, L. et al. Measuring the Symptoms of Pediatric Constipation and Irritable Bowel Syndrome with Constipation: Expert Commentary and Literature Review. Patient 7, 343–364 (2014). https://doi.org/10.1007/s40271-014-0070-3
- Irritable Bowel Syndrome
- Patient Report Outcome
- Bowel Movement
- Irritable Bowel Syndrome Patient
- Stool Consistency