Core Outcome Measures in Effectiveness Trials

Development of Core Outcome Sets for Oesophageal Atresia

General Information

Summary:
Oesophageal atresia, a congenital birth defect, affects around 1 in 3500 live births. Although survival has significantly improved to around 95%, this has revealed substantial health problems from infancy to adulthood that are still being fully defined. Most infants with OA have definitive surgical repair in the first few days of life, but in some infants the anatomy (e.g. a long gap) may preclude surgical repair of the native oesophagus, and for these the optimum oesophageal replacement (stomach, colon, jejunum) is unclear, and it is unlikely that there will ever be a randomised controlled trial to determine the optimum oesophageal replacement. Many infants with OA repaired early in life, and most with more complex anatomical variants or oesophageal replacement, will have some form of long term gastrointestinal health problems (e.g. strictures, reflux, swallowing difficulties etc.)
There are no accepted and validated outcome measures in oesophageal atresia research. This prevents accurate comparison of data, and therefore high quality research, particularly important when each paediatric surgery centre in the U.K. only encounters roughly ten cases per year, and even fewer cases in which oesophageal replacement is required. The most cited comparative studies in OA all describe a lack of heterogeneous outcome measures as a barrier to meaningful conclusions. In addition, an outcome selected purely by one group of the treating team creates implicit bias, and significant findings may therefore not be useful to patients. In order to develop robust core outcomes measures, they need to be relevant and accepted by all stakeholders.
We aim to develop two core outcome sets for Oesophageal Atresia; one for the first year of life and the second for long-term outcomes. We will engage with parent/patient support groups (TOFS, EAT), national and international organisations (BAPS, ERNICA) as stakeholder groups in order to conduct three-stage Delphi Processes in order to develop the two Core Outcome Sets.


Contributors:
Dr Simon Eaton (principal investigator)
Professor Paolo De Coppi
Mr Edward Hannon
Dr Carmen Dingemann
Professor Benno Ure
ERNICA (European Reference Network on Rare inherited and congenital diseases)

Further Study Information

Current Stage:
Ongoing
Date:
January 2018 - 2019
Funding source(s):
Currently unfunded, although supported by ERNICA (European Reference Network on Rare inherited and congenital diseases). Grant application in progress.

Health Area

Disease Category
Child health
Gastroenterology
Neonatal care

Disease Name
Oesophageal Atresia

Target Population

Age Range
0 - 100

Sex
Either


Nature / type of Intervention
Drug
Surgery

Method(s)

Consensus meeting
Delphi process
Literature review

We aim to develop two core outcome sets for Oesophageal Atresia; one for the first year of life and the second for long-term outcomes. We will engage with parent/patient support groups (TOFS, EAT), national and international organisations (BAPS, ERNICA) as stakeholder groups in order to conduct three-stage Delphi Processes in order to develop the two Core Outcome Sets.


Stakeholders Involved

Charities
Clinical experts
Consumers (patients)
Families
Patient/ support group representatives
Researchers

Study Type

COS for clinical trials or clinical research
COS for practice

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