International prospective observational study investigating the disease course and heterogeneity of paediatric-onset inflammatory bowel disease: the protocol of the PIBD-SETQuality inception cohort study

Martine A Aardoom; Polychronis Kemos; Irma Tindemans; Marina Aloi; Sibylle Koletzko; Arie Levine; Dan Turner; Gigi Veereman; Mattias Neyt; Richard K Russell; Thomas D Walters; Frank M Ruemmele; Janneke N Samsom; Nicholas M Croft; Lissy de Ridder; PIBD-SETQuality consortium and PIBD-NET

doi:10.1136/bmjopen-2019-035538

International prospective observational study investigating the disease course and heterogeneity of paediatric-onset inflammatory bowel disease: the protocol of the PIBD-SETQuality inception cohort study

BMJ Open. 2020 Jul 1;10(7):e035538. doi: 10.1136/bmjopen-2019-035538.

Authors

Martine A Aardoom¹, Polychronis Kemos², Irma Tindemans³, Marina Aloi⁴, Sibylle Koletzko^{5

6}, Arie Levine⁷, Dan Turner⁸, Gigi Veereman⁹, Mattias Neyt¹⁰, Richard K Russell¹¹, Thomas D Walters¹², Frank M Ruemmele¹³, Janneke N Samsom³, Nicholas M Croft², Lissy de Ridder¹⁴; PIBD-SETQuality consortium and PIBD-NET

Affiliations

¹ Department of Paediatric Gastroenterology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
² Centre for Immunobiology, Blizard Institute, Barts and The London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
³ Laboratory of Pediatrics, Division of Gastroenterology and Nutrition, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
⁴ Paediatric Gastroenterology and Liver Unit, Department of Paediatrics, Sapienza University of Rome, Rome, Italy.
⁵ Department of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, Ludwig Maximilians University Munich, Munich, Germany.
⁶ Department of Pediatrics, Gastroenterology and Nutrition, School of Medicine Collegium Medicum, University of Warmia and Mazury, Olsztyn, Poland.
⁷ Paediatric Gastroenterology and Nutrition Unit, Edith Wolfson Medical Center, Tel Aviv University, Holon, Israel.
⁸ Institute of Paediatric Gastroenterology, Shaare Zedek Medical Center, The Hebrew University of Jerusalem, Jerusalem, Israel.
⁹ Department of Paediatric Gastroenterology and Nutrition, UZ Brussel, Vrije Universiteit Brussel, Brussels, Belgium.
¹⁰ ME-TA Medical Evaluation and Technology Assessment, Merendree, Belgium.
¹¹ Department of Paediatric Gastroenterology, Hepatology and Nutrition, Royal Hospital for Children Glasgow, Glasgow, UK.
¹² IBD Centre, Department of Paediatrics, SickKids Hospital, University of Toronto, Toronto, Ontario, Canada.
¹³ Department of Pediatric Gastroenterology, Université Paris Descartes, Sorbonne Paris Cité, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants Malades, Paris, Île-de-France, France.
¹⁴ Department of Paediatric Gastroenterology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands L.deRidder@erasmusmc.nl.

Abstract

Introduction: Patients with paediatric-onset inflammatory bowel disease (PIBD) may develop a complicated disease course, including growth failure, bowel resection at young age and treatment-related adverse events, all of which can have significant and lasting effects on the patient's development and quality of life. Unfortunately, we are still not able to fully explain the heterogeneity between patients and their disease course and predict which patients will respond to certain therapies or are most at risk of developing a more complicated disease course. To investigate this, large prospective studies with long-term follow-up are needed. Currently, no such European or Asian international cohorts exist. In this international cohort, we aim to evaluate disease course and which patients are most at risk of therapy non-response or development of complicated disease based on patient and disease characteristics, immune pathology and environmental and socioeconomic factors.

Methods and analysis: In this international prospective observational study, which is part of the PIBD Network for Safety, Efficacy, Treatment and Quality improvement of care (PIBD-SETQuality), children diagnosed with inflammatory bowel disease <18 years are included at diagnosis. The follow-up schedule is in line with standard PIBD care and is intended to continue up to 20 years. Patient and disease characteristics, as well as results of investigations, are collected at baseline and during follow-up. In addition, environmental factors are being assessed (eg, parent's smoking behaviour, dietary factors and antibiotic use). In specific centres with the ability to perform extensive immunological analyses, blood samples and intestinal biopsies are being collected and analysed (flow cytometry, plasma proteomics, mRNA expression and immunohistochemistry) in therapy-naïve patients and during follow-up.

Ethics and dissemination: Medical ethical approval has been obtained prior to patient recruitment for all sites. The results will be disseminated through peer-reviewed scientific publications.

Trial registration number: NCT03571373.

Keywords: immunology; inflammatory bowel disease; paediatric gastroenterology.

Publication types

Observational Study
Research Support, Non-U.S. Gov't

MeSH terms

Child
Child, Preschool
Clinical Protocols*
Cohort Studies
Disease Progression
Humans
Incidence
Inflammatory Bowel Diseases
Internationality*
Prospective Studies
Quality Improvement
Risk Factors
Surveys and Questionnaires

Associated data

ClinicalTrials.gov/NCT03571373