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Congenital heart disease (CHD) is the most common birth defect in the United States. Neonates with CHD are often cared for by neonatologists in addition to cardiologists. However, there is a paucity of rigorous evidence and limited clinical trials regarding the management of neonates with CHD. In this review, we will describe some of the challenges of research in this field. The Pediatric Heart Network serves as an example of how a research network can effectively overcome barriers to conduct and execute well-designed multicenter studies., (Copyright © 2023 by the American Academy of Pediatrics.)

Background: Little is known about geographical variation in infant mortality due to congenital heart disease (CHD) and the social determinants of health that might mediate such variation. We aimed to examine US county-level estimates of infant mortality due to CHD to understand geographical patterns and factors that might influence variation in mortality., Methods: This US population-based cohort study used linked livebirth-infant death cohort files from the US National Center for Health Statistics from Jan 1, 2006, to Dec 31, 2015. All deaths attributable to congenital heart disease in infants in a given year were included. We used hierarchical Bayesian models to estimate rates of infant mortality due to congenital heart disease for all US counties. We mapped model-based estimates to explore geographical patterns. Covariates included infant sex, gestational age, maternal race and ethnicity, percentage of the county population below the poverty level, and proximity of the county to a US News & World Report 2015 top-50 ranked paediatric cardiac centre., Findings: From 2006 to 2015, 40 847 089 livebirths occurred, of which there were 13 988 infant deaths attributed to congenital heart disease, with an unadjusted infant mortality rate due to CHD of 0·34 per 1000 livebirths (95% CI 0·34-0·35). Kentucky and Mississippi had the greatest proportions of counties with a predicted rate of infant mortality due to CHD above the 95th percentile. All counties in Connecticut, Massachusetts, and Rhode Island had a predicted rate below the fifth percentile. In the model, lower mortality risk correlated with closer proximity to a top-50 ranked paediatric cardiac centre (odds ratio [OR] 0·890, 95% credible interval [CrI] 0·840-0·942), whereas higher mortality risk correlated with higher levels of poverty (OR 1·181, 95% CrI 1·125-1·239)., Interpretation: Substantial geographical variation exists in infant mortality due to CHD in the USA, highlighting the potential importance of bolstering care delivery for infants from economically deprived communities and areas remote from top-performing paediatric cardiac centres., Funding: None., Competing Interests: Declaration of interests We declare no competing interests., (Copyright © 2021 Elsevier Ltd. All rights reserved.)

Background: The Pediatric Heart Network Normal Echocardiogram Database Study had unanticipated challenges. We sought to describe these challenges and lessons learned to improve the design of future studies., Methods: Challenges were divided into three categories: enrolment, echocardiographic imaging, and protocol violations. Memoranda, Core Lab reports, and adjudication logs were reviewed. A centre-level questionnaire provided information regarding local processes for data collection. Descriptive statistics were used, and chi-square tests determined differences in imaging quality., Results: For the 19 participating centres, challenges with enrolment included variations in Institutional Review Board definitions of "retrospective" eligibility, overestimation of non-White participants, centre categorisation of Hispanic participants that differed from National Institutes of Health definitions, and exclusion of potential participants due to missing demographic data. Institutional Review Board amendments resolved many of these challenges. There was an unanticipated burden imposed on centres due to high numbers of echocardiograms that were reviewed but failed to meet submission criteria. Additionally, image transfer software malfunctions delayed Core Lab image review and feedback. Between the early and late study periods, the proportion of unacceptable echocardiograms submitted to the Core Lab decreased (14 versus 7%, p < 0.01). Most protocol violations were from eligibility violations and inadvertent protected health information disclosure (overall 2.5%). Adjudication committee reviews led to protocol changes., Conclusions: Numerous challenges encountered during the Normal Echocardiogram Database Study prolonged study enrolment. The retrospective design and flaws in image transfer software were key impediments to study completion and should be considered when designing future studies collecting echocardiographic images as a primary outcome.

Optimising short- and long-term outcomes for children and patients with CHD depends on continued scientific discovery and translation to clinical improvements in a coordinated effort by multiple stakeholders. Several challenges remain for clinicians, researchers, administrators, patients, and families seeking continuous scientific and clinical advancements in the field. We describe a new integrated research and improvement network - Cardiac Networks United - that seeks to build upon the experience and success achieved to-date to create a new infrastructure for research and quality improvement that will serve the needs of the paediatric and congenital heart community in the future. Existing gaps in data integration and barriers to improvement are described, along with the mission and vision, organisational structure, and early objectives of Cardiac Networks United. Finally, representatives of key stakeholder groups - heart centre executives, research leaders, learning health system experts, and parent advocates - offer their perspectives on the need for this new collaborative effort.

The Fontan operation creates a circulation characterized by elevated central venous pressure and low cardiac output. Over time, these characteristics result in a predictable and persistent decline in exercise performance that is associated with an increase in morbidity and mortality. A medical therapy that targets the abnormalities of the Fontan circulation might, therefore, be associated with improved outcomes. Udenafil, a phosphodiesterase type 5 inhibitor, has undergone phase I/II testing in adolescents who have had the Fontan operation and has been shown to be safe and well tolerated in the short term. However, there are no data regarding the long-term efficacy of udenafil in this population. The Fontan Udenafil Exercise Longitudinal (FUEL) Trial is a randomized, double-blind, placebo-controlled phase III clinical trial being conducted by the Pediatric Heart Network in collaboration with Mezzion Pharma Co, Ltd. This trial is designed to test the hypothesis that treatment with udenafil will lead to an improvement in exercise capacity in adolescents who have undergone the Fontan operation. A safety extension trial, the FUEL Open-Label Extension Trial (FUEL OLE), offers the opportunity for all FUEL subjects to obtain open-label udenafil for an additional 12 months following completion of FUEL, and evaluates the long-term safety and tolerability of this medication. This manuscript describes the rationale and study design for FUEL and FUEL OLE. Together, these trials provide an opportunity to better understand the role of medical management in the care of those who have undergone the Fontan operation., (Copyright © 2018 Elsevier Inc. All rights reserved.)

In an era of ongoing need for research to enable evidence-based care for the expanding population with congenital heart disease (CHD), economic fluctuations have impacted research funding. We characterize trends in NIH-funded CHD research from 2005 to 2015. We searched the NIH RePORTER database from 2005 to 2015 using the terms "congenital heart" and "cardiac morphogenesis". Projects were characterized by year, institute, mechanism, costs, type and topic, and funding trends were analyzed. From 2005 to 2015, NIH funded 633 CHD research projects with total costs of $991 million. The National Heart, Lung, and Blood Institute funded 83% of CHD projects (528, $857 million). The R01 mechanism was used for 45% of projects (288, $421 million). Projects were 70% basic/early translational research, 27% clinical research, and 3% both. Cardiac developmental biology was the most common topic (52%), followed by technology/therapy development (15%), and diagnosis/management (12%). The total number of CHD projects ranged from 153 to 221 per year (30-58 new projects/year), and costs per year ranged from $58 to $116 million. The number of projects and total costs increased until 2012, but decreased again thereafter. CHD research did not experience as much erosion as overall NIH purchasing power; in constant dollars, CHD research funding levels in 2015 were $12 million higher than those in 2005. The NIH supported a diverse portfolio of CHD projects from 2005 to 2015. Support of CHD research projects trended upward until 2012, but declined thereafter due to fiscal austerity measures.

Background: Patients with functional single ventricles after the Fontan procedure have abnormal cardiac mechanics. The aims of this study were to determine factors that influence diastolic function and to describe associations of diastolic function with current clinical status., Methods: Echocardiograms were obtained as part of the Pediatric Heart Network Fontan Cross-Sectional Study. Diastolic function grade (DFG) was assessed as normal (grade 0), impaired relaxation (grade 1), pseudonymization (grade 2), or restrictive (grade 3). Studies were also classified dichotomously (restrictive pattern present or absent). Relationships between DFG and pre-Fontan variables (e.g., ventricular morphology, age at Fontan, history of volume-unloading surgery) and current status (e.g., systolic function, valvar regurgitation, exercise performance) were explored., Results: DFG was calculable in 326 of 546 subjects (60%) (mean age, 11.7 ± 3.3 years). Overall, 32% of patients had grade 0, 9% grade 1, 37% grade 2, and 22% grade 3 diastolic function. Although there was no association between ventricular morphology and DFG, there was an association between ventricular morphology and E', which was lowest in those with right ventricular morphology (P < .001); this association remained significant when using Z scores adjusted for age (P < .001). DFG was associated with achieving maximal effort on exercise testing (P = .004); the majority (64%) of those not achieving maximal effort had DFG 2 or 3. No additional significant associations of DFG with laboratory or clinical measures were identified., Conclusions: Assessment of diastolic function by current algorithms results in a high percentage of patients with abnormal DFG, but few clinically or statistically significant associations were found. This may imply a lack of impact of abnormal diastolic function on clinical outcomes in this cohort, or it may indicate that the methodology may not be applicable to pediatric patients with functional single ventricles., (Copyright © 2016 American Society of Echocardiography. All rights reserved.)

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., (Copyright © 2016 American College of Cardiology Foundation. Published by Elsevier Inc. All rights reserved.)

Background: Collaborative learning is a technique through which individuals or teams learn together by capitalizing on one another's knowledge, skills, resources, experience, and ideas. Clinicians providing congenital cardiac care may benefit from collaborative learning given the complexity of the patient population and team approach to patient care., Rationale and Development: Industrial system engineers first performed broad-based time-motion and process analyses of congenital cardiac care programs at 5 Pediatric Heart Network core centers. Rotating multidisciplinary team site visits to each center were completed to facilitate deep learning and information exchange. Through monthly conference calls and an in-person meeting, we determined that duration of mechanical ventilation following infant cardiac surgery was one key variation that could impact a number of clinical outcomes. This was underscored by one participating center's practice of early extubation in the majority of its patients. A consensus clinical practice guideline using collaborative learning was developed and implemented by multidisciplinary teams from the same 5 centers. The 1-year prospective initiative was completed in May 2015, and data analysis is under way., Conclusion: Collaborative learning that uses multidisciplinary team site visits and information sharing allows for rapid structured fact-finding and dissemination of expertise among institutions. System modeling and machine learning approaches objectively identify and prioritize focused areas for guideline development. The collaborative learning framework can potentially be applied to other components of congenital cardiac care and provide a complement to randomized clinical trials as a method to rapidly inform and improve the care of children with congenital heart disease., (Copyright © 2016 Elsevier Inc. All rights reserved.)

Purpose of Review: Because of the relatively small numbers of pediatric patients with congenital heart disease cared for in any individual center, there is a significant need for multicenter clinical studies to validate new medical or surgical therapies. The Pediatric Heart Network (PHN), with 15 years of experience in multicenter clinical research, has tackled numerous challenges when conducting multicenter studies., Recent Findings: This review describes the challenges encountered and the strategies employed to conduct high-quality, collaborative research in pediatric cardiovascular disease., Summary: Sharing lessons learned from the PHN can provide guidance to investigators interested in conducting pediatric multicenter studies.

Background: Adolescents and young adults with congenital heart disease (CHD) are often restricted from physical activity and sports participation, which may have adverse effects., Objectives: 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., Methods: 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., Results: 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., Conclusions: Patients with CHD commonly participate in competitive sports, and such participation is associated with higher QoL, improved exercise capacity, and lower BMI., (© 2014 Wiley Periodicals, Inc.)

Objectives: The technical performance score (TPS) has been reported in a single center study to predict the outcomes after congenital cardiac surgery. We sought to determine the association of the TPS with outcomes in patients undergoing the Norwood procedure in the Single Ventricle Reconstruction trial., Methods: We calculated the TPS (class 1, optimal; class 2, adequate; class 3, inadequate) according to the predischarge echocardiograms analyzed in a core laboratory and unplanned reinterventions that occurred before discharge from the Norwood hospitalization. Multivariable regression examined the association of the TPS with interval to first extubation, Norwood length of stay, death or transplantation, unplanned postdischarge reinterventions, and neurodevelopment at 14 months old., Results: Of 549 patients undergoing a Norwood procedure, 356 (65%) had an echocardiogram adequate to assess atrial septal restriction or arch obstruction or an unplanned reintervention, enabling calculation of the TPS. On multivariable regression, adjusting for preoperative variables, a better TPS was an independent predictor of a shorter interval to first extubation (P=.019), better transplant-free survival before Norwood discharge (P<.001; odds ratio, 9.1 for inadequate vs optimal), shorter hospital length of stay (P<.001), fewer unplanned reinterventions between Norwood discharge and stage II (P=.004), and a higher Bayley II psychomotor development index at 14 months (P=.031). The TPS was not associated with transplant-free survival after Norwood discharge, unplanned reinterventions after stage II, or the Bayley II mental development index at 14 months., Conclusions: TPS is an independent predictor of important outcomes after Norwood and could serve as a tool for quality improvement., (Copyright © 2014 The American Association for Thoracic Surgery. All rights reserved.)

Purpose of Review: In fiscally challenging times, scientists must seek creative strategies and leverage existing resources to advance research., Recent Findings: This review describes programmes supported by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) to promote research in paediatric cardiology and congenital heart disease (CHD)., Summary: An understanding of NHLBI-supported research programmes will help investigators identify opportunities to collaborate with existing systems and use scientific results from existing efforts to catalyse future research in CHD.

Echocardiographic image quality in Fontan survivors may be limited by a variety of factors. We sought to describe echocardiographic quality and factors associated with study quality in subjects participating in the Pediatric Heart Network Fontan Cross-Sectional Study. Echocardiograms were obtained at 7 clinical sites using a standard protocol. Quality grading and analysis were performed by a core laboratory. Univariate and multivariable modeling were performed to assess factors associated with quality and ability to obtain images sufficient for prespecified quantitative analysis. A total of 543 echocardiograms were obtained. The quality of echocardiograms improved over the duration of the study. The great arteries, systemic veins, and pulmonary veins were less likely to be adequately imaged than other cardiac structures. Quantitative analysis of ventricular volume was possible in 76% overall, but only 41% of those with mixed ventricular morphology. Factors independently associated with better quality included younger age, levocardia, acquisition of the echocardiogram at a longer time since the beginning of enrollment, absence of a pulmonary artery stent, and clinical site. Patient and center-specific factors are associated with echocardiographic quality after the Fontan procedure. Increased familiarity and experience with a standard imaging protocol is likely to result in improved quality., (© 2013, Wiley Periodicals, Inc.)

Background and Objective: 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., Methods: 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., Results: 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., Conclusions: Screening for CCHD presents novel challenges and opportunities; however, addressing these will strengthen newborn screening and newborn care networks, and ultimately improve health outcomes.

Objectives: Identify trends of enrolment and key challenges when recruiting infants with complex cardiac diseases into a multi-centre, randomised, placebo-controlled drug trial and assess the impact of efforts to share successful strategies on enrolment of subjects., Methods: Rates of screening, eligibility, consent, and randomisation were determined for three consecutive periods of time. Sites collectively addressed barriers to recruitment and shared successful strategies resulting in the Inventory of Best Recruiting Practices. Study teams detailed institutional practices of recruitment in post-trial surveys that were compared with strategies of enrolment initially proposed in the Inventory., Results: The number of screened patients increased by 30% between the Initial Period and the Intermediate Period (p = 0.007), whereas eligibility decreased slightly by 7%. Of those eligible for entry into the study, the rate of consent increased by 42% (p = 0.025) and randomisation increased by 71% (p = 0.10). During the Final Period, after launch of a competing trial, fewer patients were screened (−14%, p = 0.06), consented (−19%, p = 0.12), and randomised (−34%, p = 0.012). Practices of recruitment in the post-trial survey closely mirrored those in the Inventory., Conclusions: Early identification and sharing of best strategies of recruitment among all recruiting sites can be effective in increasing recruitment of critically ill infants with congenital cardiac disease and possibly other populations. Strategies of recruitment should focus on those that build relationships with families and create partnerships with the medical providers who care for them. Competing studies pose challenges for enrolment in trials, but fostering trusting relationships with families can result in successful enrolment into multiple studies.

Objectives: The Single Ventricle Reconstruction trial randomized 555 subjects with a single right ventricle undergoing the Norwood procedure at 15 North American centers to receive either a modified Blalock-Taussig shunt or right ventricle-to-pulmonary artery shunt. Results demonstrated a rate of death or cardiac transplantation by 12 months postrandomization of 36% for the modified Blalock-Taussig shunt and 26% for the right ventricle-to-pulmonary artery shunt, consistent with other publications. Despite this high mortality rate, little is known about the circumstances surrounding these deaths., Methods: There were 164 deaths within 12 months postrandomization. A committee adjudicated all deaths for cause and recorded the timing, location, and other factors for each event., Results: The most common cause of death was cardiovascular (42%), followed by unknown cause (24%) and multisystem organ failure (7%). The median age at death for subjects dying during the 12 months was 1.6 months (interquartile range, 0.6 to 3.7 months), with the highest number of deaths occurring during hospitalization related to the Norwood procedure. The most common location of death was at a Single Ventricle Reconstruction trial hospital (74%), followed by home (13%). There were 29 sudden, unexpected deaths (18%), although in retrospect, 12 were preceded by a prodrome., Conclusions: In infants with a single right ventricle undergoing staged repair, the majority of deaths within 12 months of the procedure are due to cardiovascular causes, occur in a hospital, and within the first few months of life. Increased understanding of the circumstances surrounding the deaths of these single ventricle patients may reduce the high mortality rate., (Copyright © 2012 The American Association for Thoracic Surgery. All rights reserved.)

Objectives: In the Single Ventricle Reconstruction trial, infants undergoing the Norwood procedure were randomly allocated to undergo a right ventricle-to-pulmonary artery shunt or a modified Blalock-Taussig shunt. Apart from shunt type, subjects received the local standard of care. We evaluated variation in perioperative care during the Norwood hospitalization across 14 trial sites., Methods: Data on preoperative, operative, and postoperative variables for 546 enrolled subjects who underwent the Norwood procedure were collected prospectively on standardized case report forms, and variation across the centers was described., Results: Gestational age, birth weight, and proportion with hypoplastic left heart syndrome were similar across sites. In contrast, all recorded variables related to preoperative care varied across centers, including fetal diagnosis (range, 55%-85%), preoperative intubation (range, 29%-91%), and enteral feeding. Perioperative and operative factors were also variable across sites, including median total support time (range, 74-189 minutes) and other perfusion variables, arch reconstruction technique, intraoperative medication use, and use of modified ultrafiltration (range, 48%-100%). Additional variation across centers was seen in variables related to postoperative care, including proportion with an open sternum (range, 35%-100%), median intensive care unit stay (range, 9-44 days), type of feeding at discharge, and enrollment in a home monitoring program (range, 1%-100%; 5 sites did not have a program). Overall, in-hospital death or transplant occurred in 18% (range across sites, 7%-39%)., Conclusions: Perioperative care during the Norwood hospitalization varies across centers. Further analysis evaluating the underlying causes and relationship of this variation to outcome is needed to inform future studies and quality improvement efforts., (Copyright © 2012 The American Association for Thoracic Surgery. All rights reserved.)

Background: Although newborn screening for critical congenital heart disease (CCHD) was recommended by the US Health and Human Services Secretary's Advisory Committee on Heritable Disorders in Newborns and Children to promote early detection, it was deemed by the Secretary of the HHS as not ready for adoption pending an implementation plan from HHS agencies., Objective: To develop strategies for the implementation of safe, effective, and efficient screening., Methods: A work group was convened with members selected by the Secretary's Advisory Committee on Heritable Disorders in Newborns and Children, the American Academy of Pediatrics, the American College of Cardiology Foundation, and the American Heart Association., Results: On the basis of published and unpublished data, the work group made recommendations for a standardized approach to screening and diagnostic follow-up. Key issues for future research and evaluation were identified., Conclusions: The work-group members found sufficient evidence to begin screening for low blood oxygen saturation through the use of pulse-oximetry monitoring to detect CCHD in well-infant and intermediate care nurseries. Research is needed regarding screening in special populations (eg, at high altitude) and to evaluate service infrastructure and delivery strategies (eg, telemedicine) for nurseries without on-site echocardiography. Public health agencies will have an important role in quality assurance and surveillance. Central to the effectiveness of screening will be the development of a national technical assistance center to coordinate implementation and evaluation of newborn screening for CCHD.

Objectives: Although congenital heart disease is associated with low birth weight and prematurity, there is little information about these birth outcomes in infants with single ventricle physiology. We describe the birth outcomes (i.e., gestational age and birth weight) in neonates with single ventricle physiology screened for enrollment in the Pediatric Heart Network's Infant Single Ventricle Trial, compare these outcomes with US norms, and examine the association of birth outcomes with anatomic diagnosis and race., Patients and Methods: All neonates with single ventricle physiology presenting to Infant Single Ventricle Trial centers were screened for enrollment. Demographic data and anatomic diagnoses were obtained from medical records., Results: A total of 1245 neonates with single ventricle physiology were screened at 10 centers (63 to 266 per center). Diagnoses included hypoplastic left heart syndrome in 49%, unbalanced atrioventricular septal defect in 12%, and tricuspid atresia in 9%. Preterm birth occurred in 16% of neonates with single ventricle physiology vs. 12% in normal neonates (P < .001), low birth weight (<2.5 kg) in 18% vs. 8% in normals (P < .001), and small for gestational age (<10th percentile by definition) in 22% vs. 10% in normals (P < .001). A genetic syndrome was reported in 8%. The percentage of preterm birth, low birth weight, and small for gestational age was similar between screened neonates with and without hypoplastic left heart syndrome., Conclusions: In this large, contemporary cohort of neonates with single ventricle physiology, rates of preterm birth, low birth weight, and small for gestational age were higher than in the general population, but similar between screened neonates with and without hypoplastic left heart syndrome.

Most contemporary diagnostic and treatment strategies for pediatric patients with cardiovascular disease are not supported by evidence from clinical trials but instead are based on expert opinion, single-institution observational studies, or extrapolated from adult cardiovascular medicine. In response to this concern, the National Heart, Lung, and Blood Institute established the Pediatric Heart Disease Clinical Research Network (PHN) in 2001. The purposes of this article are to describe the initiation, structure, and function of the PHN; to review the ongoing studies; and to address current and future challenges. To date, four randomized clinical trials and two observational studies have been launched. Design and conduct of complex, multicenter studies in children with congenital and acquired heart disease must address numerous challenges, including identification of an appropriate clinically relevant primary endpoint, lack of preliminary data on which to base sample size calculations, and recruitment of an adequate number of subjects. The infrastructure is now well developed and capable of implementing complex, multicenter protocols efficiently and recruiting subjects effectively. The PHN is uniquely positioned to contribute to providing evidence-based medicine for and improving the outcomes of pediatric patients with cardiovascular disease.

The Working Group on research in adult congenital heart disease (ACHD) was convened in September 2004 under the sponsorship of National Heart, Lung, and Blood Institute (NHLBI) and the Office of Rare Diseases, National Institutes of Health, Department of Health and Human Services, to make recommendations on research needs. The purpose of the Working Group was to advise the NHLBI on the current state of the science in ACHD and barriers to optimal clinical care, and to make specific recommendations for overcoming those barriers. The members of the Working Group were chosen to provide expert input on a broad range of research issues from both scientific and lay perspectives. The Working Group reviewed data on the epidemiology of ACHD, long-term outcomes of complex cardiovascular malformations, issues in assessing morphology and function with current imaging techniques, surgical and catheter-based interventions, management of related conditions including pregnancy and arrhythmias, quality of life, and informatics. After research and training barriers were discussed, the Working Group recommended outreach and educational programs for adults with congenital heart disease, a network of specialized adult congenital heart disease regional centers, technology development to support advances in imaging and modeling of abnormal structure and function, and a consensus on appropriate training for physicians to provide care for adults with congenital heart disease.

Background: Live transmission of echocardiograms over integrated services digital network lines is accurate and has led to improvements in the delivery of pediatric cardiology care. Permanent archiving of the live studies has not previously been reported. Specific obstacles to permanent storage of telemedicine files have included the ability to produce accurate images without a significant increase in storage requirements., Objective: We evaluated the accuracy of Motion Pictures Expert Group (MPEG) digitization of incoming video streams and assessed the storage requirements of these files for infants in a real-time pediatric tele-echocardiography program., Results: All major cardiac diagnoses were correctly diagnosed by review of MPEG images. MPEG file size ranged from 11.1 to 182 MB (56.5 +/- 29.9 MB)., Conclusions: MPEG digitization during live neonatal telemedicine is accurate and provides an efficient method for storage. This modality has acceptable storage requirements; file sizes are comparable to other digital modalities.

To identify determinants of adverse outcome in this era of early, definitive treatment, retrospective data were analyzed for 1988 on infants aged less than 1 year with congenital cardiac disease hospitalized at The Johns Hopkins Hospital. In this cohort of 108 infants, 34% (37 of 108) had complex cardiac disease, 33% (36 of 108) had major extracardiac anomalies, 88 patients (81%) underwent 116 surgical procedures, 37% (40 of 108) were hospitalized for greater than 28 days and 29% (31 of 108) died during the first year. Univariate analysis showed that complex disease (i.e., severe ventricular hypoplasia, ventricular septal malalignment or outflow atresia), extracardiac anomalies, early initial presentation, and prolonged stay in the intensive care unit were significantly associated with infant death, whereas surgery was associated with a significantly increased rate of survival. The findings for complex disease and surgery persisted in multiple logistic regression analysis. It is concluded that outcome in most infants with congenital cardiac defects is now extremely favorable, and that major research and preventive efforts should focus on complex congenital cardiac defects.