Importance of an International Registry for and Collaborative Research on Esophageal Atresia

Abstract

Esophageal atresia (EA) is a rare congenital defect. Data on EA prevalence, management, and long-term outcome are lacking because the available data come from small retrospective series from tertiary referral centers. An international multicenter registry would provide strong epidemiological data from large population-based cohorts on EA prevalence and incidence, treatment, long-term morbidity, and prognosis and would thus provide accurate data for evaluation of the current guidelines for EA management. The future challenge of the new international network on EA, which was created in 2013, is to promote the creation of a collaborative database and further studies.

Diagnostic Workup of Neonates With Esophageal Atresia: Results From the EUPSA Esophageal Atresia Registry

“…The logical step would be to design national and international registries that may be instrumental in centralizing and collecting data to give an overall picture of the disease, which could be the basis for guidelines formation and subsequent prospective studies and randomized trials, and provide quality indicators that may be used to determine hospitals' performance and best practice variations. The importance of pediatric collaborative networks for the progress in clinical care of patients with rare disorders has been already pointed out (17)(18)(19)(20). Accordingly, the European Union is currently making an effort to develop reference networks (ERN) of expert institutions for the treatment of rare disorders, among whom ERNICA will focus specifically on congenital gastro-intestinal disorders, including EA/TEF 1 .…”

“…Accordingly, the European Union is currently making an effort to develop reference networks (ERN) of expert institutions for the treatment of rare disorders, among whom ERNICA will focus specifically on congenital gastro-intestinal disorders, including EA/TEF 1 . The INoEA (International Network on Esophageal Atresia) is another international collaboration dedicated to improving research and care for EA patients, which has the development of a collaborative database as one of its goals ( 18 ). The development of these collaborations is currently underway.…”

The French Experience with a Population-Based Esophageal Atresia Registry (RENATO)

“…20,21 Further collaboration through establishing an international registry for EA was already discussed in 2017 by the international network for EA (INoEA) and associated patient representatives. However, this concept faces challenges related to funding and compliance with the GDPR, 22 although several initiatives for international registries are currently ongoing.…”

Benchmarks for Pediatric Surgical Registries: Recommendations for the Assessment and Grading of Complications

“…However, multicentric studies on rare congenital conditions come with both advantages and disadvantages, as outlined by Gottrand et al in the context of esophageal atresia trials. 29 High-powered studies involving international collaborations and offering the prospect of publication in high impact-factor journals must contend with the long duration of such studies, the necessity to adhere to ethical/regulatory rules, the requirement to harmonize care and data collection, and the associated high cost. 29 To achieve the potential benefits of such research while mitigating the drawbacks, Arafat et al proposed that registry randomized controlled trials could represent the future of surgical studies.…”

“…29 High-powered studies involving international collaborations and offering the prospect of publication in highimpact-factor journals must contend with the long duration of such studies, the necessity to adhere to ethical/regulatory rules, the requirement to harmonize care and data collection, and the associated high cost. 29 To achieve the potential benefits of such research while mitigating the drawbacks, Arafat et al proposed that registry randomized controlled trials could represent the future of surgical studies. 30 The authors cited the examples of the Study of Access Site for Enhancement of percutaneous coronary intervention (SAFE-PCI) and Thrombus Aspiration in ST-Elevation Myocardial Infarction in Scandinavia (TASTE) trials, both in cardiology, which utilized registry data from 9,458 and 7,244 patients, respectively, for randomized controlled trials.…”