Research in Pediatric Cardiovascular Disease: NHLBI


Article title: Research in Pediatric Cardiovascular Disease: NHLBI
Conditions: Cardiovascular Disease, congenital heart defect
Source: NHLBI

National Heart, Lung, and Blood Institute
Task Force Report on

Research in Pediatric Cardiovascular Disease

May 2002

Executive Summary

Heart disease in infants, children, and adolescents is a large and under-appreciated public health problem. Diseases range from congenital structural defects present at birth to genetic abnormalities of the heart muscle and conduction system, acquired heart diseases, and adult diseases that begin in childhood. Because children have a long life ahead, the burden and cost of children’s heart disease are substantial for families and society. More than 1 million adults are alive today who had a heart defect repaired during childhood.

In January 2001, the National Heart, Lung, and Blood Institute (NHLBI) convened the Task Force on Research in Pediatric Cardiovascular Disease to identify research priorities and scientific opportunities for addressing this significant public health problem. The Task Force recommends that the NHLBI support an aggressive research program in the basic, clinical, and population sciences to capitalize on recent advances in genetics, understanding of cardiovascular development, and clinical care.

This Task Force report encompasses three broad areas: basic research on cardiovascular development and the causes of cardiovascular disease, research to improve clinical outcomes, and population strategies to reduce cardiovascular disease in adults by altering risk factors during childhood. Implementation of the Task Force's recommendations could help to foster prevention of pediatric cardiovascular disease; improve outcomes for infants, children, and adolescents with heart disease; and promote cardiovascular health among adults.

The Task Force identifies eight research priorities over the next 5 years. These priorities reflect current scientific opportunities in the following areas:

  • Fundamental studies of the formation of heart and blood vessels
  • Development and use of new and improved technologies to image the heart
  • Advanced repair of congenital heart defects in infants and children
  • Refined surgical treatment of human fetuses with heart defects
  • Exploration of stem cell biology for the repair of heart tissues
  • Creation of improved biomaterials through tissue engineering
  • Translational research to enhance clinical care
  • Definition of the childhood antecedents and risk factors for atherosclerotic heart disease in adults.

The first opportunity, cardiovascular morphogenesis, or the formation of heart and blood vessels, deserves research emphasis, to define the mechanisms of normal and abnormal morphogenesis and the role of genetic and epigenetic (environmental) factors in the development of these tissues. Basic research on these processes will yield important information on the effects of genetic and environmental risk factors and the interactions between genes and the environment in the development of heart defects. Investigators will need resources to support comparisons of genetic factors in children who have congenital heart defects with experimental models. This research could yield options for preventing heart defects.

By using advanced technologies to image the heart, researchers also could greatly improve the clinical care of children with heart defects. The Task Force recommends support of targeted programs to develop and use new and improved technologies for creating two- and three-dimensional images and assessing heart function. To accomplish this work, pediatric heart centers and technology companies must establish partnerships to develop new equipment for assessing heart disease in infants and children, to define its appropriate applications, and to determine its cost effectiveness. Use of bioinformatics is particularly important for cardiologists and surgeons who need to have real-time images of the heart combined with sophisticated measures of vascular and muscle function. Surgeons and cardiologists can enhance their repair of congenital heart defects in infants and children by using minimally invasive surgery guided by robotics, new types of therapy with catheters, and improved pre- and postsurgical support of heart and lung function. The Task Force recommends research to improve heart assist devices and technology for extracorporeal membrane oxygenation and to adapt this technology for use in infants and small children.

Surgical treatment of the human fetus is imminent. To take advantage of this opportunity to repair heart defects in utero, researchers need to develop new techniques and tools for imaging and surgically repairing the heart. Support of multicenter, collaborative studies of the natural history of heart defects in utero and the efficacy of interventions before birth would greatly help to facilitate this research.

Stem cell biology and tissue engineering also offer the potential to improve the outcomes of treatment for children with heart defects. Stem cells are a potential source of heart muscle cells and blood vessels which clinicians can use to rebuild or replace damaged heart tissue and thereby obviate the need for heart transplantation. In addition, researchers could program stem cells to produce artificial valves, blood vessels, and tissue for patches, thereby avoiding complications currently associated with the use of cardiac prosthetic materials. Establishing a clinical trials network to implement multicenter, randomized studies for rapidly assessing new therapies will accelerate the translation of research advances into clinical care. Research on clinical outcomes also is critical to implementation and development of standards of care for pediatric cardiology. Because children potentially have a long life ahead, longitudinal studies and patient registries are important for, and integral to, defining the risk of specific heart defects, appropriate surgical management, and late postoperative complications. Clinicians need to develop surrogate markers to predict optimal outcomes and identify "best practices" for various diagnoses of heart disease and across heart research centers.

Atherosclerotic heart disease in adults begins early in life. The Task Force recommends studies that define the origins of atherosclerotic vascular disease during fetal life and childhood. Development of effective therapeutic and preventive regimens depends on research strategies to identify children at high risk for future cardiovascular disease.

In this report, the Task Force details specific recommendations for each priority area. In addition, the Task Force urges action to develop and nurture an adequate, well-trained work force in academic pediatric cardiology to conduct the research needed. The future of children’s cardiovascular health depends on having an adequate work force of pediatric cardiologists, other pediatric specialists, and basic scientists to translate research findings and provide quality care. The Task Force notes concern that the number of physicians adequately prepared for these important roles is declining, at a time when the opportunity to achieve significant scientific advances is great. The Task Force urges the scientific community to devise ways to recruit and nurture the next generation of clinician scientists to conduct basic, clinical, and translational research in pediatric cardiovascular disease.

The issues and recommendations discussed by the Task Force are central to the long-term goals of understanding the causes of pediatric heart diseases, improving clinical care, and eventually preventing these diseases and their complications.

The complete report is available as a bookmarked PDF file (212 K).

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