Accepted for/Published in: JMIR Research Protocols
Date Submitted: Dec 11, 2020
Date Accepted: Apr 16, 2021
Same Same, but different? National comparative investigation in twins with congenital heart defects for their neurodevelopmental outcome and health-related quality of life: Protocol for a prospective observational study
ABSTRACT
Background:
Due to the excellent survival rates of patients with congenital heart defects (CHD), associated disorders are an increasing focus of research. Existing studies figured out an association between CHD and its treatment with neurodevelopmental outcomes including motor competence impairments. All these studies, however, compared their test results with reference values or results of healthy control groups. This comparison is influenced by socio-economic as well as genetic aspects, which do have a common known impact on neurodevelopmental outcomes.
Objective:
This study protocol describes a setting that aims to find out the role of CHD and its treatments on neurodevelopmental outcomes, excluding socioeconomic as well as genetic aspects. Only a twin comparison provides the possibility to exclude these confounding factors.
Methods:
In a German-wide prospective cohort study, 129 twin siblings registered in the National Register for Congenital Heart Defects (NRCHD) will undergo testing on cognitive function (Wechsler Intelligence Tests age-dependent WAIS-IV, WISC-V, WPPSI-IV) and motor competence (M-ABC 2). Additionally, the self-reported health-related quality of life (KINDL-R for children, SF-36 for adults), as well as parent-reported strength and difficulties of the children (SDQ-D German version) will be assessed by standardized questionnaires. CHD data on the specific diagnosis, surgical and, transcatheter procedures and, additional medical information will be received from patient records.
Results:
The approval of the Medical Ethics Committee Charité Mitte has been obtained in July 2018. After getting funded in April 2019 first enrollment was in August 2019. The study is still ongoing until June 2022. Final results are expected in 2022.
Conclusions:
This study protocol provides an overview of technical details of the study design, offering an option to exclude confounding factors on neurodevelopmental outcomes in patients with CHD. This will enable a specific analysis focusing on CHD and clinical treatments to differentiate in terms of neurodevelopmental outcomes of patients with CHD compared to twin siblings with healthy hearts. In the end, to clearly define what is important to prevent patients with CHD in terms of neurodevelopmental impairments and to define targeted prevention strategies for patients with CHD. Clinical Trial: German Clinical Trials Register: DRKS00021087; https://www.drks.de/drks_web/navigate.do?navigationId=trial.HTML&TRIAL_ID=DRKS00021087
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