Developing a core outcome set for the health outcomes for children and adults with congenital oesophageal atresia and/or tracheo-oesophageal fistula: OCELOT task group study protocol

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Abstract

Introduction

Heterogeneity in reported outcomes of infants with oesophageal atresia (OA) with or without tracheo-oesophageal fistula (TOF) prevents effective data pooling. Core outcome sets (COS) have been developed for many conditions to standardise outcome reporting, facilitate meta-analysis and improve the relevance of research for patients and families. Our aim is to develop an internationally-agreed, comprehensive COS for OA-TOF, relevant from birth through to transition and adulthood.

Methods and analysis

A long list of outcomes will be generated using (1) a systematic review of existing studies on OA-TOF and (2) qualitative research with children (patients), adults (patients) and families involving focus groups, semistructured interviews and self-reported outcome activity packs. A two-phase Delphi survey will then be completed by four key stakeholder groups: (1) patients (paediatric and adult); (2) families; (3) healthcare professionals; and (4) researchers. Phase I will include stakeholders individually rating the importance and relevance of each long-listed outcome using a 9-point Likert scale, with the option to suggest additional outcomes not already included. During phase II, stakeholders will review summarised results from phase I relative to their own initial score and then will be asked to rescore the outcome based on this information. Responses from phase II will be summarised using descriptive statistics and a predefined definition of consensus for inclusion or exclusion of outcomes. Following the Delphi process, stakeholder experts will be invited to review data at a consensus meeting and agree on a COS for OA-TOF.

Ethics and dissemination

Ethical approval was sought through the Health Research Authority via the Integrated Research Application System, registration no. 297026. However, approval was deemed not to be required, so study sponsorship and oversight were provided by Alder Hey Children’s NHS Foundation Trust. The study has been prospectively registered with the COMET Initiative. The study will be published in an open access forum.

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Most cited references 19

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A taxonomy has been developed for outcomes in medical research to help improve knowledge discovery

Susanna Dodd, Mike Clarke, Lorne Becker … (2018)

Objectives There is increasing recognition that insufficient attention has been paid to the choice of outcomes measured in clinical trials. The lack of a standardized outcome classification system results in inconsistencies due to ambiguity and variation in how outcomes are described across different studies. Being able to classify by outcome would increase efficiency in searching sources such as clinical trial registries, patient registries, the Cochrane Database of Systematic Reviews, and the Core Outcome Measures in Effectiveness Trials (COMET) database of core outcome sets (COS), thus aiding knowledge discovery. Study Design and Setting A literature review was carried out to determine existing outcome classification systems, none of which were sufficiently comprehensive or granular for classification of all potential outcomes from clinical trials. A new taxonomy for outcome classification was developed, and as proof of principle, outcomes extracted from all published COS in the COMET database, selected Cochrane reviews, and clinical trial registry entries were classified using this new system. Results Application of this new taxonomy to COS in the COMET database revealed that 274/299 (92%) COS include at least one physiological outcome, whereas only 177 (59%) include at least one measure of impact (global quality of life or some measure of functioning) and only 105 (35%) made reference to adverse events. Conclusions This outcome taxonomy will be used to annotate outcomes included in COS within the COMET database and is currently being piloted for use in Cochrane Reviews within the Cochrane Linked Data Project. Wider implementation of this standard taxonomy in trial and systematic review databases and registries will further promote efficient searching, reporting, and classification of trial outcomes.

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Core Outcome Set–STAndards for Reporting: The COS-STAR Statement

Jamie Kirkham, Sarah Gorst, Douglas G. Altman … (2016)

Background Core outcome sets (COS) can enhance the relevance of research by ensuring that outcomes of importance to health service users and other people making choices about health care in a particular topic area are measured routinely. Over 200 COS to date have been developed, but the clarity of these reports is suboptimal. COS studies will not achieve their goal if reports of COS are not complete and transparent. Methods and Findings In recognition of these issues, an international group that included experienced COS developers, methodologists, journal editors, potential users of COS (clinical trialists, systematic reviewers, and clinical guideline developers), and patient representatives developed the Core Outcome Set–STAndards for Reporting (COS-STAR) Statement as a reporting guideline for COS studies. The developmental process consisted of an initial reporting item generation stage and a two-round Delphi survey involving nearly 200 participants representing key stakeholder groups, followed by a consensus meeting. The COS-STAR Statement consists of a checklist of 18 items considered essential for transparent and complete reporting in all COS studies. The checklist items focus on the introduction, methods, results, and discussion section of a manuscript describing the development of a particular COS. A limitation of the COS-STAR Statement is that it was developed without representative views of low- and middle-income countries. COS have equal relevance to studies conducted in these areas, and, subsequently, this guideline may need to evolve over time to encompass any additional challenges from developing COS in these areas. Conclusions With many ongoing COS studies underway, the COS-STAR Statement should be a helpful resource to improve the reporting of COS studies for the benefit of all COS users.

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Oesophageal atresia: prevalence, prenatal diagnosis and associated anomalies in 23 European regions.

Elisa Calzolari, Steffen Husby, Ester Garne … (2012)

To describe prevalence, prenatal diagnosis and epidemiological data on oesophageal atresia from 23 well-defined European regions and compare the prevalence between these regions. Population-based study using data from a large European database for surveillance of congenital anomalies (EUROCAT) for two decades (1987-2006). Twenty-three participating registries based on multiple sources of information including information about live births, fetal deaths with gestational age ≥20 weeks and terminations of pregnancy. 1222 cases of oesophageal atresia in a population of 5 019 804 births. The overall prevalence was 2.43 cases per 10 000 births (95% CI 2.30 to 2.57). There were regional differences in prevalence ranging from 1.27 to 4.55. Prenatal detection rates varied by registry from >50% of cases to <10% of cases. A total of 546 cases (44.7%) had an isolated oesophageal anomaly, 386 (31.6%) were multiple malformed and 290 (23.7%) had an association or a syndrome. There were 1084 live born cases (88.7%), 43 cases were fetal deaths and 95 cases were terminations of pregnancy. One-week survival for live births was 86.9% and 99.2% if the gestational age was ≥38 weeks and isolated oesophageal atresia was present. Males accounted for 57.3% of all cases and 38.5% of live born cases were born with gestational age <37 weeks. There were regional differences in prevalence of oesophageal atresia in Europe. Half of all cases had associated anomalies. Prenatal detection rate increased from 26% to 36.5% over the two decades. Survival in infants with isolated oesophageal atresia born at term is high.

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Author and article information

Journal

Journal ID (nlm-ta): BMJ Paediatr Open

Journal ID (iso-abbrev): BMJ Paediatr Open

Journal ID (hwp): bmjpo

Journal ID (publisher-id): bmjpo

Title: BMJ Paediatrics Open

Publisher: BMJ Publishing Group (BMA House, Tavistock Square, London, WC1H 9JR )

ISSN (Electronic): 2399-9772

Publication date Collection: 2024

Publication date (Electronic): 5 February 2024

Volume: 8

Issue: 1

Electronic Location Identifier: e002262

Affiliations

[1 ]departmentDepartment of Paediatric and Neonatal Surgery , Ringgold_4593Alder Hey Children's NHS Foundation Trust , Liverpool, Merseyside, UK

[2 ]departmentDepartment of Paediatric and Neonatal Surgery , Ringgold_105554Royal Manchester Children's Hospital , Manchester, UK

[3 ]departmentDivision of Developmental Biology and Medicine, Faculty of Biology Medicine and Health , Ringgold_5292The University of Manchester , Manchester, UK

[4 ]departmentDepartment of Health Data Science , Ringgold_4591University of Liverpool , Liverpool, UK

[5 ]MRC/NIHR Trials Methodology Research Partnership , Liverpool, UK

[6 ]departmentEvidence-based Practice Research Centre , Ringgold_6249Edge Hill University , Ormskirk, UK

[7 ]departmentChildren's Nursing Research Unit , Ringgold_4593Alder Hey Children's NHS Foundation Trust , Liverpool, Merseyside, UK

[8 ]departmentDepartment of Pediatric Surgery , Ringgold_97759Erasmus MC Sophia Children Hospital , Rotterdam, Zuid-Holland, The Netherlands

[9 ]departmentDepartment of Paediatric and Neonatal Surgery , Ringgold_59841Royal Hospital for Children and Young People , Edinburgh, UK

[10 ]departmentDepartment of Dietetics , Ringgold_8741Yeovil District Hospital NHS Foundation Trust , Yeovil, UK

[11 ]departmentDepartment of Clinical Psychology , Ringgold_4593Alder Hey Children's NHS Foundation Trust , Liverpool, Merseyside, UK

[12 ]departmentDepartment of Pediatric Surgery , Sor Maria Ludovica Hospital , La Plata, Argentina

[13 ]TOFS , Nottingham, UK

[14 ]departmentDepartment of Speech and Language Therapy , Ringgold_2153Cambridge University Hospitals NHS Foundation Trust , Cambridge, UK

[15 ]departmentDepartment of Respiratory Medicine , Ringgold_4472Leeds Teaching Hospitals NHS Trust , Leeds, UK

[16 ]departmentDepartment of Pediatric Surgery , Ringgold_9177Hannover Medical School , Hannover, Germany

[17 ]departmentDepartment of ENT Surgery , Ringgold_4593Alder Hey Children's NHS Foundation Trust , Liverpool, Merseyside, UK

[18 ]departmentDepartment of Paediatric , Ringgold_8903Cardiff and Vale University Health Board , Cardiff, UK

[19 ]departmentDepartment of Physiotherapy , Ringgold_4593Alder Hey Children's NHS Foundation Trust , Liverpool, Merseyside, UK

[20 ]departmentDepartment of Paediatric and Neonatal Surgery , Ringgold_2153Cambridge University Hospitals NHS Foundation Trust , Cambridge, UK

[21 ]departmentDepartment of Paediatric Surgery , Ringgold_26685Mahidol University , Salaya, Thailand

[22 ]Ringgold_12303University of Cincinnati College of Medicine , Cincinnati, Ohio, USA

[23 ]departmentFaculty of Medicine and Health Sciences , Ringgold_121470Stellenbosch University , Cape Town, South Africa

[24 ]departmentPaediatric Surgery , Ringgold_6453The Royal Children's Hospital Melbourne , Melbourne, Victoria, Australia

[25 ]Ringgold_34361Murdoch Children's Research Institute , Parkville, Victoria, Australia

[26 ]departmentPediatrics, Division of Respirology , Ringgold_27338Children's Hospital of Eastern Ontario , Ottawa, Ontario, Canada

[27 ]departmentPediatrics , Ringgold_6363University of Ottawa , Ottawa, Ontario, Canada

[28 ]departmentDepartment of Pediatric Gastroenterology , Ringgold_371501Sydney Children's Hospitals Network , Westmead, New South Wales, Australia

[29 ]departmentDepartment of Surgery , Ringgold_37447University of Malaya , Kuala Lumpur, Malaysia

[30 ]departmentDepartment of Paediatric Surgery , Ringgold_37579KK Women's and Children's Hospital , Singapore

[31 ]departmentDepartment of Paediatric Surgery , Ringgold_6453The Royal Children's Hospital Melbourne , Melbourne, Victoria, Australia

[32 ]Murdoch Children’s Research Institute , Melbourne, Victoria, Australia

[33 ]departmentDepartment of Pediatrics, College of Medicine , Ringgold_12303University of Cincinnati , Cincinnati, Ohio, USA

[34 ]departmentUniversity Surgery Unit, Faculty of Medicine , Ringgold_7423University of Southampton , Southampton, UK

[35 ]departmentRespiratory Unit , Ringgold_4593Alder Hey Children's NHS Foundation Trust , Liverpool, UK

Author notes

[Correspondence to ] Dr Rebecca Thursfield; rebecca.thursfield@ 123456alderhey.nhs.uk

Author information

Lucy Bray http://orcid.org/0000-0001-8414-3233

Nadine Teunissen http://orcid.org/0000-0003-4474-2930

Paul Cullis http://orcid.org/0000-0003-4479-6621

Nigel J Hall http://orcid.org/0000-0001-8570-9374

Rebecca Thursfield http://orcid.org/0000-0002-5793-685X

Article

Publisher ID: bmjpo-2023-002262

DOI: 10.1136/bmjpo-2023-002262

PMC ID: 10860107

PubMed ID: 38316469

SO-VID: cf4a42fd-a659-44e7-9d34-073e0e98be7b

License:

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, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.

History

Date received : 25 September 2023

Date accepted : 03 December 2023

Funding

Funded by: Alder Hey Children’s Charity;

Award ID: N/A

Funded by: TOFS charity;

Award ID: N/A

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Keywords: data collection,gastroenterology,infant,neonatology

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Keywords: data collection, gastroenterology, infant, neonatology

Developing a core outcome set for the health outcomes for children and adults with congenital oesophageal atresia and/or tracheo-oesophageal fistula: OCELOT task group study protocol

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Abstract

Introduction

Methods and analysis

Ethics and dissemination

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Karger: Gastroenterology

Most cited references 19

A taxonomy has been developed for outcomes in medical research to help improve knowledge discovery

Core Outcome Set–STAndards for Reporting: The COS-STAR Statement

Oesophageal atresia: prevalence, prenatal diagnosis and associated anomalies in 23 European regions.

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