Clinical research priorities in adult congenital heart disease
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.
References (155)
- et al.
Multicenter research in adult congenital heart disease
Int J Cardiol
(2008) - et al.
Optimal timing for pulmonary valve replacement in adults after tetralogy of Fallot repair
Am J Cardiol
(2005) - et al.
Impact of pulmonary valve replacement in tetralogy of Fallot with pulmonary regurgitation: a comparison of intervention and nonintervention
Ann Thorac Surg
(2012) - et al.
Factors associated with impaired clinical status in long-term survivors of tetralogy of Fallot repair evaluated by magnetic resonance imaging
J Am Coll Cardiol
(2004) - et al.
Left ventricular dysfunction is a risk factor for sudden cardiac death in adults late after repair of tetralogy of Fallot
J Am Coll Cardiol
(2002) - et al.
Risk factors for arrhythmia and sudden cardiac death late after repair of tetralogy of Fallot: a multicentre study
Lancet
(2000) The adult with congenital heart disease: born to be bad?
J Am Coll Cardiol
(2005)- et al.
Canadian Cardiovascular Society 2009 Consensus Conference on the management of adults with congenital heart disease: outflow tract obstruction, coarctation of the aorta, tetralogy of Fallot, Ebstein anomaly and Marfan's syndrome
Can J Cardiol
(2010) Repaired tetralogy of Fallot: the roles of cardiovascular magnetic resonance in evaluating pathophysiology and for pulmonary valve replacement decision support
J Cardiovasc Magn Reson
(2011)- et al.
Pulmonary valve replacement after operative repair of tetralogy of Fallot. Meta-analysis and meta-regression of 3118 patients from 48 studies
J Am Coll Cardiol
(2013)
Long-term survival in patients with repair of tetralogy of Fallot: 36-year follow-up of 490 survivors of the first year after surgical repair
J Am Coll Cardiol
A population-based prospective evaluation of risk of sudden cardiac death after operation for common congenital heart defects
J Am Coll Cardiol
Sustained ventricular tachycardia in adult patients late after repair of tetralogy of Fallot
J Am Coll Cardiol
Significance of early transient complete heart block as a predictor of sudden death late after operative correction of tetralogy of Fallot
Am J Cardiol
Left and right ventricular diastolic function in adults with surgically repaired tetralogy of Fallot: a multi-institutional study
Can J Cardiol
Right ventricular function in asymptomatic individuals with a systemic right ventricle
J Am Soc Echocardiogr
Subjective evaluation of right ventricular systolic function in hypoplastic left heart syndrome: how accurate is it?
J Am Soc Echocardiogr
Long-term outcome in congenitally corrected transposition of the great arteries: a multi-institutional study
J Am Coll Cardiol
Systemic right ventricular longitudinal strain is reduced in adults with transposition of the great arteries, relates to subpulmonary ventricular function, and predicts adverse clinical outcome
Am Heart J
The role of echocardiography in the assessment of right ventricular systolic function in patients with transposition of the great arteries and atrial redirection
Arch Cardiovasc Dis
Transverse strain predicts exercise capacity in systemic right ventricle patients
Int J Cardiol
Right ventricular function with standard and speckle-tracking echocardiography and clinical events in adults with D-transposition of the great arteries post atrial switch
J Am Soc Echocardiogr
Dynamic assessment of right ventricular volumes and function by real-time three-dimensional echocardiography: a comparison study with magnetic resonance imaging in 100 adult patients
J Am Soc Echocardiogr
Clinical value of real-time three-dimensional echocardiography for right ventricular quantification in congenital heart disease: validation with cardiac magnetic resonance imaging
J Am Soc Echocardiogr
Three-dimensional echocardiographic assessment of right ventricular volume and function in adult patients with congenital heart disease: comparison with magnetic resonance imaging
J Am Soc Echocardiogr
Echocardiographic knowledge-based reconstruction for quantification of the systemic right ventricle in young adults with repaired D-transposition of great arteries
Am J Cardiol
Evaluating the systemic right ventricle by CMR: the importance of consistent and reproducible delineation of the cavity
J Cardiovasc Magn Reson
Long-term outcome in congenitally corrected transposition of the great arteries: a multi-institutional study
J Am Coll Cardiol
Arrhythmia and mortality after the Mustard procedure: a 30-year single-center experience
J Am Coll Cardiol
Congenitally corrected transposition of the great arteries ventricular function at the time of systemic atrioventricular valve replacement predicts long-term ventricular function
J Am Coll Cardiol
Functional outcome of patients operated for d-transposition of the great arteries with the Mustard procedure
Am J Cardiol
Outcome of the unoperated adult who presents with congenitally corrected transposition of the great arteries
J Am Coll Cardiol
Arrhythmia and mortality after the Mustard procedure: a 30-year single-center experience
J Am Coll Cardiol
Late results of Senning operation
J Thorac Cardiovasc Surg
Results of a multicenter retrospective implantable cardioverter-defibrillator registry of pediatric and congenital heart disease patients
J Am Coll Cardiol
Predictors of sudden cardiac death after Mustard or Senning repair for transposition of the great arteries
J Am Coll Cardiol
Clinical correlation and prognostic predictive value of neurohumoral factors in patients late after the Fontan operation
Am Heart J
ACC/AHA 2008 Guidelines for the Management of Adults with Congenital Heart Disease: a report of the American College of Cardiology/American Heart Association Task Force on Practice Guidelines (writing committee to develop guidelines on the management of adults with congenital heart disease)
Circulation
Comparison of shunt types in the Norwood procedure for single-ventricle lesions
N Engl J Med
Tight glycemic control versus standard care after pediatric cardiac surgery
N Engl J Med
CONCOR, an initiative towards a national registry and DNA-bank of patients with congenital heart disease in the Netherlands: rationale, design, and first results
Eur J Epidemiol
Indications for pulmonary valve replacement in repaired tetralogy of Fallot: the quest continues
Circulation
Long-term outcome in patients undergoing surgical repair of tetralogy of Fallot
N Engl J Med
Long-term results of total repair of tetralogy of Fallot in adulthood: 35 years follow-up in 104 patients corrected at the age of 18 or older
Thorac Cardiovasc Surg
Ventricular size and function assessed by cardiac MRI predict major adverse clinical outcomes late after tetralogy of Fallot repair
Heart
Randomized trial of pulmonary valve replacement with and without right ventricular remodeling surgery
Circulation
Biventricular response after pulmonary valve replacement for right ventricular outflow tract dysfunction: is age a predictor of outcome?
Circulation
Pulmonary valve replacement in tetralogy of Fallot: impact on survival and ventricular tachycardia
Circulation
Outcome of pulmonary valve replacements in adults after tetralogy repair: a multi-institutional study
Congenit Heart Dis
Variability in surgical referral patterns for pulmonary valve replacement in adults with repaired tetralogy of Fallot
Congenit Heart Dis
Cited by (31)
The Future of Adult Congenital Heart Disease Research: Precision Health Services Delivery for the Next Decade
2019, Canadian Journal of CardiologyHeart Failure in Adults With Congenital Heart Disease
2018, Heart Failure in the Child and Young Adult: From Bench to BedsidePulmonary Valve Procedures Late After Repair of Tetralogy of Fallot: Current Perspectives and Contemporary Approaches to Management
2017, Canadian Journal of CardiologyCitation Excerpt :Further detail regarding evaluation of the patient with rTOF are in the Evaluation of the Patient With Repaired Tetralogy of Fallot section of the Supplementary Material. The ongoing, as yet unresolved, controversy pertaining to the management of chronic PR has been identified as a top research priority in adult congenital heart disease.31 As the links between chronic severe PR, RV dilatation, and dysfunction and late adverse events have become more defined there has been heightened concern surrounding appropriate referral strategies for PVR.
Heart Failure in Adults With Congenital Heart Disease
2017, Heart Failure in the Child and Young Adult: From Bench to BedsideEmerging Research Directions in Adult Congenital Heart Disease A Report from an NHLBI/ACHA Working Group
2016, Journal of the American College of CardiologyThree parties, one direction: Research priorities in adults with congenital heart disease. What do professionals, patients and relatives want to know?
2016, International Journal of CardiologyCitation Excerpt :Assessing the importance assigned to individual research topics from both a medical and the patients' point of view should allow for future research to be specifically geared to the patients' needs without losing sight of the medical facts. Knowing patients', relatives' and physicians' view regarding the importance of different research topics should also help designing targeted studies, the results of which will most probably find their way into clinical routine, while considering aspects of time and cost efficiency [1]. Moreover, the assessment of the future need for research sheds light on the implicit need of information regarding medical and psychosocial issues.