Outcomes have improved significantly in pediatric cardiovascular disease in recent decades. The challenge now is to sustain these advances through innovative clinical trials, fundamental molecular ...investigations, genetics and genomics, and outreach to families, emphasizing the importance of participating in research. We describe several such efforts and provide a vision of the future for pediatric cardiovascular research.
Critical congenital heart disease (CCHD) is endorsed by the US Secretary of Health and Human Services as part of the recommended uniform screening panel for newborns. Although initial recommendations ...for implementation exist, as states and hospitals have moved forward with implementation of screening, new challenges and areas for additional focus have been identified. The objective of this study was to develop recommendations to address current challenges and areas of focus surrounding CCHD newborn screening.
A workgroup of experts and stakeholders was convened in Washington, District of Columbia, for a 1-day meeting in February 2012. At the beginning of the meeting, the stakeholders held a brainstorming session to identify areas of main priority based on their experience. After this, stakeholders broke into small groups to refine recommendations, which were then finalized by consensus.
Recommendations to address selection of screening equipment, standards for reporting of screening outcomes to stakeholders, training of health care providers and educating families, future research priorities, payment for screening, follow-up diagnostic testing, and public health oversight, and advocacy to facilitate effective and comprehensive screening were proposed. Suggestions for future work were developed.
Screening for CCHD presents novel challenges and opportunities; however, addressing these will strengthen newborn screening and newborn care networks, and ultimately improve health outcomes.
Adolescents and young adults with congenital heart disease (CHD) are often restricted from physical activity and sports participation, which may have adverse effects.
To determine the amount of ...physical activity, type of sports participation, and reasons for sports restrictions, and to evaluate the effect of sports participation on quality of life (QoL) in a cohort of patients with CHD.
Individuals with CHD aged 13-30 years were recruited at outpatient visits or via mailings. They completed a questionnaire addressing physical activity, sports participation, sports restrictions, and QoL (Pediatric Quality of Life Inventory). We also reviewed the patient's medical record.
Of the 177 patients who responded (mean age 20 years), 31% have mild CHD, 40% have moderate CHD, and 29% have severe CHD. In the cohort, 52% participate in competitive sports, 25% recreational sports, and 23% no sports. Among patients with severe CHD, 29% participate in competitive sports that would be restricted by published guidelines (36th Bethesda Conference). After controlling for age, sex, CHD severity, residual hemodynamic disease, and comorbidities, participation in competitive sports and increased frequency of physical activity are independently associated with a higher QoL (P = .003 and P = .001, respectively). In an identical model, competitive sports participation and frequency of physical activity are associated with higher maximum predicted oxygen consumption (VO2 ) (n = 40; P = .002 and .02) and slightly lower body mass index (BMI) (P = .02 and .01). All findings were similar when analyses were stratified by recruitment method.
Patients with CHD commonly participate in competitive sports, and such participation is associated with higher QoL, improved exercise capacity, and lower BMI.
Abstract 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. 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 adult CHD. 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.
Educating Parents About Pediatric Research Marceau, Lisa D.; Welch, Lisa C.; Pemberton, Victoria L. ...
Qualitative health research,
07/2016, Volume:
26, Issue:
8
Journal Article
Peer reviewed
Open access
A gap in information about pediatric clinical trials exists, and parents remain uncertain about what is involved in research studies involving children. We aimed to understand parent perspectives ...about pediatric clinical research after viewing the online Children and Clinical Studies (CaCS) program. Using a qualitative descriptive study design, we conducted focus groups with parents and phone interviews with physicians. Three themes emerged providing approaches to improve parent’s understanding of clinical research by including strategies where parents (a) hear from parents like themselves to learn about pediatric research, (b) receive general clinical research information to complement study-specific details, and (c) are provided more information about the role of healthy child volunteers. Parents found the website a valuable tool that would help them make a decision about what it means to participate in research. This tool can assist parents, providers, and researchers by connecting general information with study-specific information.
...many of the clinical registries in pediatric and congenital heart disease share clinical nomenclature for disease classification and procedural coding. During early childhood, a child is more ...likely to have multiple visits to the physician’s office, and children see multiple specialists throughout childhood. ...there are indeed many characteristics of pediatric care that differ from adult care. Name Employment Consultant Speaker Ownership/Partnership/Principal Research Institutional, Organizational, or Other Financial Benefit Expert Witness Jeffrey R. Boris (Chair) Children’s Hospital of Philadelphia-Clinical Professor of Pediatrics, Division of Cardiology None None None None None None Marie J. Béland Montreal Children’s Hospital None None None None None None Lisa J. Bergensen Boston Children’s Hospital-Associate in Cardiology None None None None None None Steven D. Colan Boston Children’s Hospital-Director of Clinical Research, Cardiology None None None None None None Joanna Dangel Medical University of Warsaw-Professor of Pediatric Cardiology None None None None None None Curtis J. Daniels Nationwide Children’s-Director Adolescent and Adult Congenital Heart Disease None None None None None None Christopher Davis Rady Children’s Hospital San Diego-Director, Cardiopulmonary Exercise Laboratory None None None None None None Allen D. Everett Johns Hopkins All Children’s Hospital-Director of the Pediatric Proteome Center None None None None None None Rodney Franklin Royal Brompton Hospital-Consultant Paediatric Cardiologist None None None None None None J. William Gaynor Children’s Hospital of Philadelphia-Director, Fetal Neuroprotection and Neuroplasticity Program None None None None None None Darryl T. Gray Agency for Healthcare Research and Quality-Medical Officer None None None None None None Jennifer C. Hirsch-Romano University of Michigan-Associate Director, Pediatric Cardiothoracic Intensive Care Unit None None None None None None Jeffrey P. Jacobs Johns Hopkins All Children’s Hospital-Chief, Division of Cardiovascular Surgery None None None None None None Marshall Jacobs Johns Hopkins Cardiac Surgery-Director, Pediatric Cardiac Surgery Outcomes Research None None None None None None Howard Jeffries Seattle Children’s Hospital-Medical Director, Clinical Effectiveness None None None None None None Otto Nils Krogmann Klinik Für Kinderkardiologie None None None None None None Edwin A. Lomotan Agency for Healthcare Research and Quality-Medical Officer None None None None None None Leo Lopez Nicklaus Children’s Hospital-Medical Director, Noninvasive Cardiac Imaging None None None None None None Ariane Marelli McGill University Health Centre-Associate Professor of Medicine None None None None None None Gerard R. Martin Children’s National Health System-Medical Director of Global Health None None None None None None G. Paul Matherne University of Virginia Children’s Hospital-Associate Chief Medical Officer None None None None None None Constantine Mavroudis Johns Hopkins Children’s Heart Surgery-Site Director None None None None None None Ken McCardle Mount Sinai Health System-Senior Director, Clinical Operations None None None None None None Gail D. Pearson National Heart, Lung, and Blood Institute-Director, Adult and Pediatric Cardiac Research Program None None None None None None Geoffrey Rosenthal University of Maryland-Director, Children’s Heart Program None None None None None None John S. Scott Office of the Assistant Secretary of Defense, Health Affairs-Program Director, Clinical Informatics Policy None None None None None None Gerald A. Serwer C.S. Mott Children’s Hospital-Professor, Pediatrics None None None None None None Stephen S. Seslar Seattle Children’s Hospital-Associate Professor, Pediatric Cardiology None None None None None None Robert Shaddy Chief Professor-Children’s Hospital of Philadelphia None None None None None None Timothy Slesnick Emory University School of Medicine-Assistant Professor of Pediatrics None None None None None None David F. Vener Texas Children’s Hospital-Associate Professor, Pediatrics and Anesthesiology None None None None None None Henry L. Walters III Children’s Hospital of Michigan-Chief, Cardiovascular Surgery None None None None None None Paul M. Weinberg Children’s Hospital of Philadelphia-Director, Cardiac Registry None None None None None None This table represents the relationships of committee members with industry and other entities that were determined to be relevant to this document. Meaningful Use Definition & Objectives (15) Current every day smoker Current some day smoker Former smoker Never smoker Smoker, current status unknown Unknown if ever smoked Heavy tobacco smoker Light tobacco smoker Illicit Drug Use The nonmedical use of chemicals that are prohibited by international law. listlist_itemYes/list_itemlist_itemNo/list_itemlist_itemUnknown/list_item/list World Health Organization (34) ▪CABG indicates coronary artery bypass graft; CAD, coronary artery disease; CHD, congenital heart disease; IEP, individualized education plan; NCI, National Cancer Institute; NICHD, National Institute of Child Health and Human Development; MI, myocardial infarction; and PCI, percutaneous coronary intervention.BMI indicates body mass index; BSA, body surface area; BP, blood pressure, mmHg, millimeter mercury; and RA, right arm.
An important aspect of translational research is that the flow of knowledge along the continuum is in both directions. ...observations from ongoing clinical trials may suggest potential areas of ...inquiry for the developmental and genetic researchers, and assist in prioritizing aspects of the animal studies that have the most clinical relevance. Institution Project Title Cardiovascular Development Consortium Gladstone Institutes The Epigenetic Landscape of Heart Development Harvard University Mapping Transcriptional Networks in Cardiac Development University of Pittsburgh Modeling the Genetic Basis for Human Congenital Heart Disease in Mice University of Utah Genome-wide Analysis of Cardiac Development in Zebrafish Pediatric Cardiac Genomics Consortium Children's Hospital Boston/Brigham and Women's Hospital Copy Number Variants for Discovery of Congenital Heart Genes Children's Hospital of Philadelphia The Genetic Basis of Conotruncal Defects Columbia University Molecular Approaches to Gene Identification in Congenital Heart Disease Mount Sinai School of Medicine Genomic Studies of Secundum Atrial Septal Defects Yale University Genetic Determinants of Human Heterotaxy and Aortic Arch Malformation Table 1 Institutions and Project Titles of the Cardiovascular Development and Pediatric Cardiac Genomics Consortia
Abstract Background In children with single right ventricular (RV) anomalies, changes in RV size and function may be influenced by shunt type chosen at the time of the Norwood procedure. Objectives ...The study sought to identify shunt-related differences in echocardiographic findings at 14 months and ≤6 months pre-Fontan in survivors of the Norwood procedure. Methods We compared 2-dimensional and Doppler echocardiographic indices of RV size and function, neo-aortic and tricuspid valve annulus dimensions and function, and aortic size and patency at 14.1 ± 1.2 months and 33.6 ± 9.6 months in subjects randomized to a Norwood procedure using either the modified Blalock-Taussig shunt (MBTS) or right ventricle to pulmonary artery shunt (RVPAS). Results Acceptable echocardiograms were available at both time points in 240 subjects (114 MBTS, 126 RVPAS). At 14 months, all indices were similar between shunt groups. From the 14-month to pre-Fontan echocardiogram, the MBTS group had stable indexed RV volumes and ejection fraction, while the RVPAS group had increased RV end-systolic volume (p = 0.004) and decreased right ventricular ejection fraction (RVEF) (p = 0.004). From 14 months to pre-Fontan, the treatment groups were similar with respect to decline in indexed neo-aortic valve area, >mild neo-aortic valve regurgitation (<5% at each time), indexed tricuspid valve area, and ≥moderate tricuspid valve regurgitation (<20% at each time). Conclusions Initial Norwood shunt type influences pre-Fontan RV remodeling during the second and third years of life in survivors with single RV anomalies, with greater RVEF deterioration after RVPAS. Encouragingly, other indices of RV function remain stable before Fontan regardless of shunt type. (Comparison of Two Types of Shunts in Infants with Single Ventricle Defect Undergoing Staged Reconstruction—Pediatric Heart Network; NCT00115934 )
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