Clinical research priorities in adult congenital heart disease

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Abstract

Background

Adult congenital heart disease (ACHD) clinicians are hampered by the paucity of data to inform clinical decision-making. The objective of this study was to identify priorities for clinical research in ACHD.

Methods

A list of 45 research questions was developed by the Alliance for Adult Research in Congenital Cardiology (AARCC), compiled into a survey, and administered to ACHD providers. Patient input was sought via the Adult Congenital Heart Association at community meetings and online forums. The 25 top questions were sent to ACHD providers worldwide via an online survey. Each question was ranked based on perceived priority and weighted based on time spent in ACHD care. The top 10 topics identified are presented and discussed.

Results

The final online survey yielded 139 responses. Top priority questions related to tetralogy of Fallot (timing of pulmonary valve replacement and criteria for primary prevention ICDs), patients with systemic right ventricles (determining the optimal echocardiographic techniques for measuring right ventricular function, and indications for tricuspid valve replacement and primary prevention ICDs), and single ventricle/Fontan patients (role of pulmonary vasodilators, optimal anticoagulation, medical therapy for preservation of ventricular function, treatment for protein losing enteropathy). In addition, establishing criteria to refer ACHD patients for cardiac transplantation was deemed a priority.

Conclusions

The ACHD field is in need of prospective research to address fundamental clinical questions. It is hoped that this methodical consultation process will inform researchers and funding organizations about clinical research topics deemed to be of high priority.

Introduction

Adult congenital heart disease (ACHD) clinicians are hampered by the paucity of robust data to inform clinical decision-making. The American College of Cardiology/American Heart Association 2008 Guidelines for the Management of Adults with Congenital Heart Disease have been an important effort in standardizing ACHD care, although limited by a lack of strong evidence to support many of the recommendations. The document consists of 513 individual recommendations, of which 5 (0.97%) are based on level A evidence (multiple randomized trials or meta-analyses), 161 (31.4%) based on level B evidence (a single randomized trial or nonrandomized studies), and 347 (67.6%) based on level C evidence (expert opinion, case studies, or standards of care) [1].

To date, the vast majority of multi-center research studies in ACHD have been descriptive or observational. However, newer efforts including notable examples from pediatric cardiology and congenital cardiac surgery have demonstrated the feasibility of prospective, randomized trials [2], [3]. The development of large, nationwide registries, such as the Dutch CONCOR registry and others has contributed importantly to our understanding of prevalence and natural history of CHD [4]. The Alliance for Adult Research in Congenital Cardiology (AARCC) has also pooled resources to complete multicenter studies [5]. As prospective, randomized trials are expensive and time-intensive, careful prioritization of potential study topics is prudent.

Given scarce resources for executing studies, it is worthwhile to focus on topics with the greatest potential to positively impact clinical management. Therefore, the aim of this study was to methodically pool opinions of key stakeholders, including both patients and providers, in identifying top priorities in clinical ACHD research.

Section snippets

Methods

Initially, a list of potential clinical research questions was generated through “brainstorming” sessions by AARCC investigators based on a list of congenital heart defects and general topics to ensure comprehensive consideration of all lesions. Distinct and specific research questions were generated for each, rather than general issues. Questions were circulated and revised over several iterations during a 12-month period. Overlapping questions were consolidated. From this initial list,

Results

A total of 57 surveys were received from the Toronto conference. Respondents included 43 MD/DO, 8 RN, 5 NP/PA, and 1 other. Of these, the time spent in ACHD care was > 75% for 44% of respondents, 50–75% for 19% of respondents, 25–50% for 12% of respondents, and < 25% for 23% of respondents. The top 25 ranked questions that were further considered are listed (Table 2).

The online survey yielded 139 responses. Of these 50 (36%) reported > 75% of time spent in ACHD care, with 15%, 27%, and 22% of

Discussion

While no list can incorporate all potential important research questions of interest, our objective was to highlight research topics deemed to be of high priority by a methodical consultation process of relevant stakeholders. Each of the top 10 research questions is discussed in greater detail, in random order, below.

Conclusions

The ACHD field is in need of dedicated prospective research to address fundamental questions in clinical management. We sought to consolidate and provide some focus on the myriad of research topics that could be pursued, while fostering patient–physician partnerships. It is hoped that this list will spark interest and inspire researchers and funding organizations to pursue required studies in the field and address important care-limiting issues.

Acknowledgment

This project was sponsored by a grant from the National Heart, Lung and Blood Institute (RC4 HL104831) via the American Recovery and Reinvestment Act of 2009. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Heart, Lung and Blood Institute of the National Institutes of Health.

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