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 childrens 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
childrens 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).
Basic Information
on PDF files Screen Readers and PDF
Trouble Opening
PDF files
|