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Original article
Development of a core outcome set for clinical trials in childhood constipation: a study using a Delphi technique
  1. Sophie Kuizenga-Wessel1,
  2. Nina Francesca Steutel1,
  3. Marc Alexander Benninga1,
  4. Thierry Devreker2,
  5. Elena Scarpato3,
  6. Annamaria Staiano3,
  7. Hania Szajewska4,
  8. Yvan Vandenplas2,
  9. Merit Monique Tabbers1
  1. 1 Department of Paediatric Gastroenterology and Nutrition, Emma Children’s Hospital, Academic Medical Centre, Amsterdam, The Netherlands
  2. 2 Department of Paediatrics, Free University of Brussels, Brussels, Belgium
  3. 3 Department of Translational Medical Sciences - Section of Paediatrics, University of Naples Federico II, Naples, Italy
  4. 4 Department of Paediatrics, The Medical University of Warsaw, Warsaw, Poland
  1. Correspondence to Ms Nina Francesca Steutel; n.f.steutel{at}amc.uva.nl

Abstract

Objective Patients, their parents and healthcare professionals (HCPs) have a different perception regarding the symptoms of functional constipation (FC). Consequently, a lack of agreement exists on definitions and outcomes used in therapeutic trials of FC. Therefore, our aim was to develop a core outcome set (COS) for FC for children aged 0–1 year and 1–18 years.

Design and setting Prospective study design: primary, secondary and tertiary care settings.

Methods This COS was developed using a Delphi technique. First, HCPs, parents of children with FC and patients aged ≥12–18 years were asked to list up to five outcomes they considered relevant in the treatment of FC. Outcomes mentioned by >10% of participants were included in a shortlist. In the next phase, outcomes on this shortlist were rated and prioritised by HCPs, parents and patients. Outcomes with the highest scores were included in a draft COS. In a face-to-face expert meeting, the final COS was determined.

Results The first phase was completed by 109 HCPs, 165 parents and 50 children. Fifty HCPs, 80 parents and 50 children completed the subsequent phase. The response rate was between 63% and 100% in both steps. The final COS for all ages consisted of: defecation frequency, stool consistency, painful defecation, quality of life, side effects of treatment, faecal incontinence, abdominal pain and school attendance.

Conclusion The use of this COS for FC will decrease study heterogeneity and improve comparability of studies. Therefore, researchers are recommended to use this COS in future therapeutic trials on childhood FC.

  • outcomes measures
  • patient reported outcomes
  • parent reported outcomes
  • paediatrics
  • functional constipation

This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/

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Footnotes

  • Contributors SK-W: participated in the design of the study, collected data, performed data analyses, drafted the initial manuscript and approved the final manuscript as submitted. NFS: participated in the design of the study, collected data, performed data analyses, drafted the initial manuscript and approved the final manuscript as submitted. MAB: participated in the design of the study, supervised drafting of the manuscript and approved the final manuscript as submitted. TD: collected data, participated in writing the manuscript and approved the final manuscript as submitted. ES: collected data, participated in writing the manuscript and approved the final manuscript as submitted. AS: participated in the design of the study, collected data and approved the final manuscript as submitted. YV: participated in the design of the study, collected data and approved the final manuscript as submitted. HS: participated in the design of the study, collected data and approved the final manuscript as submitted. MMT: participated in the design of the study, supervised drafting of the manuscript and approved the final manuscript as submitted.

  • Competing interests None declared.

  • Patient consent Obtained.

  • Provenance and peer review Commissioned; externally peer reviewed.

  • Data sharing statement Tables with reported outcome measures of step 2 are available from the authors.

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