Background: Despite anatomical correction, people born with oesophageal atresia±tracheoesophageal fistula (OA-TOF) experience lifelong morbidity. Core outcome sets (COSs) are recognised as a means of improving research quality and, as a consequence, improving patient outcomes; one was not available for this population.
Objective: The scope of the study was to develop a COS for people born with OA-TOF that would be applicable regardless of age or geographic location.
Study design: Patient input was paramount to this study. For long-list generation, in addition to the systematic review (SR), patients and representatives were invited to participate in focus groups, interviews or complete activity packs to ascertain outcomes that matter most to them. International consensus was then sought using a two-step Delphi survey followed by an online consensus meeting.
Results: Eight outcomes were identified through patient events that had not been picked up from SR. 175 people completed the Delphi survey from 26 countries and health care professionals from 13 different disciplines. 24 outcomes met predefined criteria for inclusion and following discussion and voting in the consensus meeting, and 14/24 outcomes were agreed for inclusion in the COS.
Conclusion: 14 outcomes have been agreed on to form the COS. 12 of these outcomes are relevant to people of all ages, 1 to paediatric population and 1 to adult cohorts. The COS is, therefore, truly applicable lifelong, which was the scope of the project. This COS will help reduce research heterogeneity, enabling better quality research outcomes and more comparable data.
Babies born with oesophageal atresia (OA); - a gap in their swallowing food pipe meaning no connection between their mouth and stomach; and tracheoesophageal fistula (TOF); - an abnormal connection between food pipe and wind pipe; have this lesion corrected with emergency life saving surgery. Despite surgical operation, functional problems remain for many TOF patients causing significant symptoms throughout childhood and into adulthood. With an incidence of 1 in 3,500 births this rare congenital disorder is surprisingly under-researched and there are few comprehensive robust data on long-term outcomes beyond single centre reports.
We plan develop ‘core outcomes’ by working nationally, linking with key healthcare professionals and importantly including patient and parent representation(s) groups. These core outcome(s) will then be used to establish a database / registry for longitudinal data to be studied and archived. A registry will permit robust analysis at a UK national population level making a real impact towards improving health outcomes for all TOF children.
Rebecca Thursfield, Sarah Gorst, Nadine Teunisson, Nick Lansdale, Julia Faulkner, Usha Krishnan, Tom Kovesi, Graham Slater, Paul Cullis, Lucy Bray, A Donne, Warwick Teague, Paul D Losty, Siobhan Carr, Victoria Gray, Lucia Gutierrez-Gammino, Shireen Anne Nah, Nigel J Hall
Disease Category: Ear, nose, & throat, Gastroenterology
Disease Name: Oesophageal Atresia, Tracheoesophageal Fistula
Age Range: 0 - 120
Sex: Either
Nature of Intervention: Surgery
- Charities
- Clinical experts
- Families
- Patient/ support group representatives
- Service users
- COS for clinical trials or clinical research
- COS for registry
- Recommendations for outcome measures (measurement/how)
- Consensus meeting
- Delphi process
- Focus group(s)
- Interview
- Literature review
- Survey
- Systematic review
Using systematic reviews, parent/patient interviews, questions on social media and focus groups we will identify a list of all possible outcomes. We will use social media platforms to get a wide range of opinions from the affected community and then focus groups, each looking at specific and differing areas will refine these ideas. The resulting possible outcomes will be refined and important outcomes elicited using a Delphi process involving the patient community and healthcare professionals. The COS will be finalised in a consensus meeting, again involving both PPI representatives and healthcare professionals.