Clinical research priorities in adult congenital heart disease

Cotts, Timothy B.; Khairy, Paul; Opotowsky, Alexander R.; John, Anitha S.; Valente, Anne Marie; Zaidi, Ali N.; Cook, Stephen C.; Aboulhosn, Jamil; Ting, Jennifer; Gurvitz, Michelle; Landzberg, Michael J.; Verstappen, Amy; Kay, Joseph; Earing, Michael G.; Franklin, Wayne; Kogon, Brian; Broberg, Craig S.

doi:10.1016/j.ijcard.2013.12.034

Cited by 36 publications

(22 citation statements)

References 147 publications

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“…Patients are starting to partner with investigators to identify research priorities that are relevant to their experience. 11 In some cases, patients and their advocacy groups are establishing their own research initiatives, recruiting patients into registries, designating centers of excellence, and educating regulatory agencies about how best to support their community's needs.…”

Section: Engaging Patientsmentioning

confidence: 99%

Perspective on Congenital Heart Disease Research

Kaltman

Burns

Pearson

2017

Circulation Research

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C ongenital heart disease (CHD) is the most common birth defect and a leading cause of morbidity and mortality in patients with congenital malformations. The past 7 decades have witnessed dramatic advances in the medical, catheter, and surgical management of these patients. Clinical research has been a driving force to improve our understanding of the pathophysiology of disease and to refine therapy. Here, we identify some of the themes that have characterized the research process in the past and that have propelled the field to its present state. We discuss current research-related challenges and speculate about what trends may define the future of CHD research.The historical canon of congenital heart disease (CHD) is replete with dramatic triumphs: the first open heart surgery, the first use of cardiopulmonary bypass, the first transcatheter interventional cardiac procedure, and many others. In the years since these seminal events, progress has been made in all aspects of the care for patients with CHD. Prompt diagnosis is routine, surgical management of the most complex lesions is achievable, and acute and chronic care of patients has been refined. Important challenges remain, including persistent mortality for particular complex defects and long-term problems such as neurodevelopmental abnormalities, arrhythmias, and heart failure. Nevertheless, there are many reasons to be proud, and clinical research has been a foundational feature that has helped drive much of this progress. We describe some trends in research that have propelled the field to its current state and speculate about what trends may define the future of CHD research. Past and Present CHD Research Individual to Group ScienceA defining trend of the past 75 years of CHD research has been the change in focus from the individual to the group.Early CHD research was performed by individuals trying to solve problems, 1 child at a time. Innovative thinkers and doers-Gross, Lillehei, Rashkind, and Norwood-pushed the envelope, often in the face of opposition, to expand the realm of the possible. This early research is perhaps best exemplified by the first blue baby operation. Helen Taussig, a pediatric cardiologist at Johns Hopkins, systematically studied these babies and concluded that inadequate flow to the pulmonary artery was the cause of their hypoxemia. She convinced the surgeon, Alfred Blalock, and his collaborator, Vivien Thomas, to devise a surgical strategy to augment pulmonary blood flow. Working in the dog laboratory, Blalock and Thomas perfected the technique of anastomosing the subclavian artery to the pulmonary artery. Their systematic study led to one of the first translational research accomplishments in CHD, the first successful blue baby operation performed in 1944. No multicenter clinical trial was needed to confirm the enormous benefit of this operation, and the principle has proven remarkably durable for children with inadequate pulmonary blood flow.This and other early, innovative approaches to management of CHD had tremendous eff...

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Section: Engaging Patientsmentioning

confidence: 99%

Perspective on Congenital Heart Disease Research

Kaltman

Burns

Pearson

2017

Circulation Research

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“…On the basis of expert opinion and published research priorities in CHD (20,21), 3 broad high-impact areas were identified: HF; vascular disease; and multisystem complications. The first interest area involved understanding the pathophysiology of and optimizing management strategies for HF in CHD, a common problem that will continue to grow as the ACHD population ages.…”

Section: Working Groupmentioning

confidence: 99%

“…Over the past decade, significant progress has been made toward these goals (19-21), due to the support of advocacy groups and federal agencies, increased training of ACHD clinician-scientists, and volunteerism of professionals and societies. The Alliance for Adult Research in Congenital Cardiology (22), a research network of investigators from 15 centers, was created in 2006 (19).…”

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confidence: 99%

Emerging Research Directions in Adult Congenital Heart Disease

Gurvitz

Burns

Brindis

et al. 2016

Journal of the American College of Cardiology

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Congenital heart disease (CHD) is the most common birth defect, affecting about 0.8% of live births. Advances in recent decades have allowed >85% of children with CHD to survive to adulthood, creating a growing population of adults with CHD. Little information exists regarding survival, demographics, late outcomes, and comorbidities in this emerging group, and multiple barriers impede research in adult CHD (ACHD). The National Heart, Lung, and Blood Institute and the Adult Congenital Heart Association convened a multidisciplinary Working Group to identify high-impact research questions in ACHD. This report summarizes the meeting discussions in the broad areas of CHD-related heart failure, vascular disease and multisystem complications. High-priority subtopics identified included heart failure in tetralogy of Fallot, mechanical circulatory support/transplantation, sudden cardiac death, vascular outcomes in coarctation of the aorta, late outcomes in single ventricle disease, cognitive and psychiatric issues, and pregnancy.

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“…Cotts et al for the Alliance for Adult Research in Congenital Cardiology compiled a list of 45 questions and conducted a survey of providers to identify 10 priority questions, with some input from patient groups. [19] Of these, four were specific to patients with single ventricle heart conditions: ‘Is pulmonary vasodilator therapy beneficial in Fontan patients?’, ‘Are warfarin and/or aspirin beneficial in preventing primary thromboembolic events in adult Fontan patients?’, ‘What is the optimal medical therapy for preservation of ventricular systolic and diastolic function in Fontan patients?’ and ‘What is the optimal medical treatment algorithm for Fontan patients with protein-losing enteropathy (PLE)?’, whilst another was also relevant: ‘What are the ideal criteria for transplantation referral in ACHD?’. Similar questions to each of these were posed in our initial survey but only preservation of ventricular function was included as a priority.…”

Section: Discussionmentioning

confidence: 99%

Research priorities in single-ventricle heart conditions: a United Kingdom national study

et al. 2018

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Objective To bring together stakeholders in the United Kingdom to establish national priorities for research in single ventricle heart conditions. Methods This study comprised two surveys and a workshop. The initial public online survey asked respondents for up to 3 questions they would like answered by research. Responses were classified as: unanswered, already answered, or unable to be answered by scientific research. In the follow-up survey, unanswered questions were divided into categories and respondents were asked to rank categories and questions by priority. A stakeholder workshop attended by patients, parents, healthcare professionals, researchers and charities was held to determine a final list of research priorities. Results 128 respondents posed 344 research questions, of which 271 were classified as unanswered, and after removing duplicates, 204 questions remained, divided into 20 categories. 56 (49.1%) respondents successfully ranked categories and questions in the second survey. 39 participants attended the workshop, drawing up a list of 30 research priorities across 9 priority categories: Associated co-morbidities, Brain & neurodevelopment, Exercise, Fontan failure, Heart function, Living with a single ventricle heart condition, Management of the well-functioning Fontan circulation, Surgery & perioperative care, and Transplantation, mechanical support & novel therapies. Conclusions Through a multi-stage process, we engaged a wide range of interested parties to establish a list of research priorities in single ventricle heart conditions. This provides a platform for clinicians, researchers and funders in the UK and elsewhere to address the most important questions and improve outcomes in these rare but high-impact congenital heart defects.

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Clinical research priorities in adult congenital heart disease

Cited by 36 publications

References 147 publications

Perspective on Congenital Heart Disease Research

Perspective on Congenital Heart Disease Research

Emerging Research Directions in Adult Congenital Heart Disease

Research priorities in single-ventricle heart conditions: a United Kingdom national study

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