Development and Validation of a Core Outcome Set for Adolescents and Young Adults with Congenital Heart Diseases (AYAs-CHD COS) addressing Transition to Adult Care: A Delphi Study

Adolescents and Young Adults (AYAs) with congenital heart disease (CHD) represent a growing population facing significant disability, morbidity, and frailty. As they reach adulthood, their clinical, psychosocial, and educational needs evolve, making the transition to adult care a critical and delicate phase for both them and their caregivers. To address these lifelong challenges, structured transitional programmes are essential. Consequently, there is a growing body of knowledge focused on transition to adult care and transitional programmes tailored to AYAs with CHD and their families' needs. Transitional programmes include multidimensional transition clinic (TC) interventions structured into different phases (i.e., pre-transition, transition, and post-transition) and tailored to age, clinical status, and social needs.

To date, outcome measurement in this field has been guided by two main international standard sets: (a) the ICHOM (i.e., International Consortium for Health Outcomes Measurement) standard set for CHD developed by Hummel et al. (2021), addressing both adult and pediatric outcomes and encompassing Patient Reported Outcome Measures (PROMs) from four macro-areas (i.e., overall health, mental health, physical health and social health); the Core Outcome Set (COS) focused on adolescents and young adults (AYAs) with special health care needs (SHCN) developed by Fair et al. (2016), which includes individual outcomes (e.g., quality of life and adherence to medication), social outcomes (e.g., having a social network), and health services outcomes (e.g., attending medical appointments) during the health care transition. Both datasets' scope is broad, in terms of care – as the dataset by Hummel et al. (2021) refers to the overall care pathway and not specifically to TC interventions, and in terms of population – as the COS by Fair et al. (2016) refers to AYAs with SHCN and not specifically with CHD. While foundational, these international sets were not developed to specifically measure outcomes sensitive to TC programmes in the CHD population.

This scenario reveals the need to reach a consensus on a synthesis of core outcomes to provide evidence of the effectiveness of the TC interventions on AYAs with CHD and their families. This study aims to fill this gap by developing and validating a specific COS for AYAs with CHD (AYAs-CHD COS) and their families (i.e., caregivers/parents), addressing the transition from pediatric to adult care within the Italian context. This COS will be developed through a rigorous Delphi consensus process. It will synthesise overlapping outcomes established by Hummel et al. (2021) and Fair et al. (2016) and complement them with new, specific outcomes sensitive to the Italian TC model of care. The scope of the resulting COS will cover outcomes sensitive to TC interventions provided to AYAs with CHD (all types) and families. The reference framework will be the Transition Framework by Moons et al. (2021), which outlines the transitional phases and the proposed timing for implementation. The resulting COS will provide a standardised, nationally validated tool for both research and clinical quality improvement, ensuring that the impact of Italian transition services is measured comprehensively and consistently.

Contributors

Angileri S., PhD(s) RN, Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
Maga G., PhD, RM, Department of Public Health, Experimental and Forensic Medicine, Section of Hygiene, University of Pavia, Pavia, Italy
Ciolfi D., PhD, RN, Pediatric and Transition Cardiology, Meyer Children's Hospital – IRCCS, Florence, Italy
Severi F., PhD (s), RN, Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
Magon A., PhD, RN, Health Professions Research and Development Unit, IRCCS San Donato, San Donato Milanese
Flocco S., MSN, RN, Health Professions Research and Development Unit, IRCCS San Donato, San Donato Milanese
Caruso R., FAAN, PhD, RN, Health Professions Research and Development Unit, IRCCS San Donato, San Donato Milanese, Italy & Department of Biomedical Sciences for Health, University of Milan, Milan, Italy
De Maria M., PhD, RN, Department of Life Sciences, Health, and Health Professions, Link Campus University, Rome, Italy

Further Study Information

Current Stage: Ongoing
Date: May 2025 - 2026
Funding source(s): No funding sources


Health Area

Disease Category: Heart & circulation

Disease Name: Congenital heart disease

Target Population

Age Range: 12 - 29

Sex: Either

Nature of Intervention: Any

Stakeholders Involved

- Clinical experts
- Policy makers
- Service users

Study Type

- COS for clinical trials or clinical research
- COS for practice

Method(s)

- Delphi process
- Literature review
- Nominal group technique (NGT)
- Systematic review

A multi-method and multi-phase study will be conducted, consistent with the recommendations of the “COnsensus-based Standards for the selection of health Measurement Instruments” (COMET) Handbook: version 1.0 by Williamson et al. (2017), the Conducting and REporting DElphi Studies (CREDES) guidance by Jünger et al. (2017), and the ACcurate COnsensus Reporting Document (ACCORD) guideline by Gattrell et al. (2014). The Developmental phase will aim to outline a preliminary version of the AYAs-CHD COS, based on the literature and the scientific evidence. Potential core outcomes will be extracted from the key literature and the guidelines on the topic. Then, the steering committee will conduct a face-to-face meeting to refine the preliminary version of the COS using the nominal group technique (NGT) method. The Consensus Process phase will aim to reach a consensus on the COS through the Delphi technique involving three distinct panels, each consisting of selected stakeholders (i.e., clinical experts, policy makers, and service users) to ensure a balance of expertise and experience.

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