Evaluation and Management of Chronic Heart Failure in Children and Adolescents With Congenital Heart Disease: A Scientific Statement From the American Heart Association.

With continued medical and surgical advancements, most children and adolescents with congenital heart disease are expected to survive to adulthood. Chronic heart failure is increasingly being recognized as a major contributor to ongoing morbidity and mortality in this population as it ages, and treatment strategies to prevent and treat heart failure in the pediatric population are needed. In addition to primary myocardial dysfunction, anatomical and pathophysiological abnormalities specific to various congenital heart disease lesions contribute to the development of heart failure and affect potential strategies commonly used to treat adult patients with heart failure. This scientific statement highlights the significant knowledge gaps in understanding the epidemiology, pathophysiology, staging, and outcomes of chronic heart failure in children and adolescents with congenital heart disease not amenable to catheter-based or surgical interventions. Efforts to harmonize the definitions, staging, follow-up, and approach to heart failure in children with congenital heart disease are critical to enable the conduct of rigorous scientific studies to advance our understanding of the actual burden of heart failure in this population and to allow the development of evidence-based heart failure therapies that can improve outcomes for this high-risk cohort.

Patient and Device Selection in Pediatric MCS: A Review of Current Consensus and Unsettled Questions.

The field of pediatric ventricular assist device (VAD) support has expanded significantly over the past 20 years, with one third of pediatric heart transplant recipients currently being bridged to transplant with a VAD. Despite increased pediatric VAD utilization, however, there remains little formalized guidance for patient or device selection. The population of children with advanced heart failure is quite heterogeneous, and the available data suggest that VAD outcomes vary significantly based upon patient size, anatomy, level of illness, and type of device implanted. In an effort to better understand current practice patterns and identify populations for whom there does not appear to be a consensus approach to achieving optimal VAD outcomes, the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) has surveyed clinical decision-making among member sites and conducted a review of the available literature regarding patient population-specific VAD outcomes and center-specific practices related to patient and device selection. Rather than aiming to provide clinical guidelines, this document offers an overview of contemporary approaches to patient and device selection, highlighting specific populations for whom there is not a consensus approach to achieving reliably good VAD outcomes, as these populations may benefit most from future research and quality improvement efforts directed toward identifying best practice.

A change of heart: Preliminary results of the US 2018 adult heart allocation revision.

In 2018, the Organ Procurement and Transplantation Network (OPTN) modified adult heart allocation to better stratify candidates and provide broader access to the most medically urgent candidates. We analyzed OPTN data that included waiting list and transplant characteristics, geographical distribution, and early outcomes 1 year before (pre: October 18, 2017-October 17, 2018) and following (post: October 18, 2018-October 17, 2019) implementation. The number of adult heart transplants increased from 2954 pre- to 3032 postimplementation. Seventy-eight percent of transplants in the post era were for the most medically urgent (statuses 1-3) compared to 68% for status 1A in the pre era. The median distance between the donor hospital and transplant center increased from 83 to 216 nautical miles, with an increase in total ischemic time from 3 to 3.4 hours (all P < .001). Waiting list mortality was not different across eras (14.8 vs 14.9 deaths per 100 patient-years pre vs post respectively). Posttransplant patient survival was not different, 93.6% pre and 92.8% post. There is early evidence that the heart allocation policy has enhanced stratification of candidates by their medical urgency and broader distribution for the most medically urgent candidates with minimal impact on overall waiting list mortality and posttransplant outcomes.

Heart transplantation in an infant with Williams-Beuren syndrome and rapidly progressive ischemic cardiomyopathy.

Ischemic cardiomyopathy with resultant refractory HF may occur in patients with WBS, often as the result of coronary involvement with SVAS. The rapid development of arteriopathy at a young age raises concerns regarding transplant candidacy due to progressive stenoses at other arterial sites with potential detrimental impact on long-term heart graft function. We report a 2-month-old male infant diagnosed with mild aortic stenosis during the neonatal period, but subsequently developed rapidly progressive supravalvar and coronary artery stenoses leading to cardiogenic shock due to myocardial ischemia. The presentation led to the diagnosis of WBS. He required prolonged CPR including ECMO therapy. He subsequently underwent LVAD implantation as bridge to transplant and 4 days later heart transplantation. His post-operative course was complicated by prolonged mechanical ventilation and extended intensive care unit and hospital stays. However, at follow-up 18 months post-transplant he continues to have normal graft function with mild, non-progressive residual coarctation of aorta and non-progressive moderately hypoplastic pulmonary arteries.

A comprehensive strategy in donor acceptance: Impact on pediatric waitlist and heart transplant outcomes.

Significant inter- and intra-center practice variability is present in pediatric donor heart acceptability. This may contribute to variation in the donor refusal rate and may impact waitlist time, morbidity, mortality, and transplant rates. In order to reduce practice variability, our center developed and implemented a comprehensive strategy regarding donor acceptance in September 2017. The aim of this study was to assess the impact of this strategy on waitlist time and outcomes as well as early post-transplant outcomes. We performed a single-center, retrospective analysis of all pediatric (<18 years) patients listed for single-organ heart transplant at our center from September 2015 to September 2018. Patients were divided into those listed before (Group 1) and after implementation of the comprehensive strategy (Group 2). The primary end-point was waitlist time. Secondary end-points included waitlist removal due to death or clinical deterioration, donor refusals per listed patient, early post-transplant outcomes (graft failure, mechanical ventilation time, inotropic support, length of hospital stay) and 1-year post-transplant survival. Of 78 listed patients, 54 were transplanted (29 in Group 1), 9 were removed due to death or clinical deterioration (7 in Group 1) and 15 were removed due to clinical improvement (12 in Group 1). The waitlist time was significantly shorter in Group 2 (17 days, IQR 7-53) vs Group 1 (90 days, IQR 14-162); P = .006. The number of donor refusals was lower in Group 2 (1, IQR 0-2.2) vs Group 1 (4, IQR 2-19); P < .001. The percentage of refused donors with normal function (Left ventricular ejection fraction > 50%) was lower in Group 2 vs Group 1 (53% vs 84%; P < .001). Difference in removal from the waitlist for death or deterioration in Group 2 vs Group 1 (n = 2, 7% vs n = 7, 20%, P = .18) did not reach statistical significance. There was no difference in post-transplant outcomes between groups. The waitlist time and donor refusals significantly decreased after implementation of a comprehensive donor acceptance strategy without impacting transplant outcomes. This analysis supports the need for a comprehensive approach to donor organ acceptance within a pediatric transplant center.

Review of the discard and/or refusal rate of offered donor hearts to pediatric waitlisted candidates.

We aimed to review current literature on the discard rate of donor hearts offered to pediatric recipients and assess geographical differences. Consequences and ways to reduce the discard rate are discussed. A systemic review on published literature on pediatric transplantation published in English since 2010 was undertaken. Additionally, a survey was sent to international OPOs with the goal of incorporating responses from around the world providing a more global picture. Based on the literature review and survey, there is a remarkably wide range of discard and/or refusal for pediatric hearts offered for transplant, ranging between 18% and 57% with great geographic variation. The data suggest that that the overall refusal rate may have decreased over the last decade. Reasons for organ discard were difficult to identify from the available data. Although the refusal rate of pediatric donor hearts seems to be lower compared to that reported in adults, it is still as high as 57% with geographic variation.

Review of interactions between high-risk pediatric heart transplant recipients and marginal donors including utilization of risk score models.

BACKGROUND: Donor organ acceptance practices vary among pediatric heart transplant professionals. We sought to understand what is known about the interactions between the "high-risk" recipient and the "marginal donor," and how donor risk scores can impact this discussion. METHODS: A systematic review of published literature on pediatric HTx was undertaken with the assistance of a medical librarian. Two authors independently assessed search results, and papers were reviewed for inclusion. RESULTS: We found that there are a large number of individual factors, and clusters of factors, that have been used to label individual recipients "high-risk" and individual donors "marginal." The terms "high-risk recipient" and "marginal donor" have been used broadly in the literature making it virtually impossible to make comparisons between publications. In general, the data support that patients who could be easily agreed to be "sicker recipients" are at more risk compared to those who are clearly "healthier," albeit still "sick enough" to need transplantation. Given this variability in the literature, we were unable to define how being a "high-risk" recipient interplays with accepting a "marginal donor." Existing risk scores are described, but none were felt to adequately predict outcomes from factors available at the time of offer acceptance. CONCLUSIONS: We could not determine what makes a donor "marginal," a recipient "high-risk," or how these factors interplay within the specific recipient-donor pair to determine outcomes. Until there are better risk scores predicting outcomes at the time of organ acceptance, programs should continue to evaluate each organ and recipient individually.

Review of the impact of donor characteristics on pediatric heart transplant outcomes.

Heart transplantation (HTx) is a treatment option for end-stage heart failure in children. HTx is limited by the availability and acceptability of donor hearts. Refusal of donor hearts has been reported to be common with reasons for refusal including preexisting donor characteristics. This review will focus on the impact of donor characteristics and comorbidities on outcomes following pediatric HTx. A literature review was performed to identify articles on donor characteristics and comorbidities and pediatric HTx outcomes. There are many donor characteristics to consider when accepting a donor heart. Weight-based matching is the most common form of matching in pediatric HTx with a donor-recipient weight ratio between 0.7 and 3 having limited impact on outcomes. From an age perspective, donors <50 years can be carefully considered, but the impact of ischemic time needs to be understood. To increase the donor pool, with minimal impact on outcomes, ABO-incompatible donors should be considered in patients that are eligible. Other factors to be considered when accepting an organ is donor comorbidities. Little is known about donor comorbidities in pediatric HTx, with most of the data available focusing on infections. Being aware of the potential infections in the donor, understanding the testing available and risks of transmission, and treatment options for the recipient is essential. There are a number of donor characteristics that potentially impact outcomes following pediatric HTx, but these need to be taken into consideration along with their interactions with recipient factors when interpreting the outcomes following HTx.

Pediatric cardiac waitlist mortality-Still too high.

Cardiac transplantation for children with end-stage cardiac disease with no other medical or surgical options is now standard. The number of children in need of cardiac transplant continues to exceed the number of donors considered "acceptable." Therefore, there is an urgent need to understand which recipients are in greatest need of transplant before becoming "too ill" and which "marginal" donors are acceptable in order to reduce waitlist mortality. This article reviewed primarily pediatric studies reported over the last 15 years on waitlist mortality around the world for the various subgroups of children awaiting heart transplant and discusses strategies to try to reduce the cardiac waitlist mortality.

Pediatric donor management to optimize donor heart utilization.

Optimal management of pediatric cardiac donors is essential in order to maximize donor heart utilization and minimize the rate of discarded organs. This review was performed after a systematic literature review and gives a detailed overview on current practices and guidelines. The review focuses on optimal monitoring of pediatric donors, donor workup, hormonal replacement, and obliterating the adverse effects of brain death. The current evidence on catecholamine support and thyroid hormone replacement is also discussed. Recognizing and addressing this shall help in a standardized approach toward donor management and optimal utilization of pediatric heart donors organs.

Effects of donor cause of death, ischemia time, inotrope exposure, troponin values, cardiopulmonary resuscitation, electrocardiographic and echocardiographic data on recipient outcomes: A review of the literature.

BACKGROUND: Heart transplantation has become standard of care for pediatric patients with either end-stage heart failure or inoperable congenital heart defects. Despite increasing surgical complexity and overall volume, however, annual transplant rates remain largely unchanged. Data demonstrating pediatric donor heart refusal rates of 50% suggest optimizing donor utilization is critical. This review evaluated the impact of donor characteristics surrounding the time of death on pediatric heart transplant recipient outcomes. METHODS: An extensive literature review was performed to identify articles focused on donor characteristics surrounding the time of death and their impact on pediatric heart transplant recipient outcomes. RESULTS: Potential pediatric heart transplant recipient institutions commonly receive data from seven different donor death-related categories with which to determine organ acceptance: cause of death, need for CPR, serum troponin, inotrope exposure, projected donor ischemia time, electrocardiographic, and echocardiographic results. Although DITs up to 8 hours have been reported with comparable recipient outcomes, most data support minimizing this period to <4 hours. CVA as a cause of death may be associated with decreased recipient survival but is rare in the pediatric population. Otherwise, however, in the setting of an acceptable donor heart with a normal echocardiogram, none of the other data categories surrounding donor death negatively impact pediatric heart transplant recipient survival. CONCLUSIONS: Echocardiographic evaluation is the most important donor clinical information following declaration of brain death provided to potential recipient institutions. Considering its relative importance, every effort should be made to allow direct image visualization.

Behavioral economics-A framework for donor organ decision-making in pediatric heart transplantation.

The high discard rate of pediatric donor hearts presents a major challenge for children awaiting heart transplantation. Recent literature identifies several factors that contribute to the disparities in pediatric donor heart usage, including regulatory oversight, the absence of guidelines on pediatric donor heart acceptance, and variation among transplant programs. However, a likely additional contributor to this issue are the behavioral factors influencing transplant team decisions in donor offer scenarios, a topic that has not yet been studied in detail. Behavioral economics and decision psychology provide an excellent foundation for investigating decision-making in the pediatric transplant setting, offering key insights into the behavior of transplant professionals. We conducted a systematic review of published literature in pediatric heart transplant related to behavioral economics and the psychology of decision-making. In this review, we draw on paradigms from these two domains in order to examine how existing aspects of the transplant environment, including regulatory oversight, programmatic variation, and allocation systems, may precipitate potential biases surrounding donor offer decisions. Recognizing how human decision behavior influences donor acceptance is a first step toward improving utilization of potentially viable pediatric donor hearts.

Early Surgical Closure of Atrial Septal Defect Improves Clinical Status of Symptomatic Young Children with Underlying Pulmonary Abnormalities.

Elective closure of atrial septal defect (ASD) is usually recommended during preschool ages. However, ASD may contribute to deteriorating health in the presence of significant comorbidity and, thus, may need earlier closure. There is a lack of clarity regarding the indications for and outcomes after ASD closure in infancy and early childhood. We investigated the benefits and safety of surgical ASD closure in symptomatic patients under 2 years of age. Retrospective chart review was conducted in patients who underwent surgical ASD closure within the first 2 years of life. Of 31 symptomatic ASD patients, 22 had persistent respiratory symptoms, 24 failure to thrive, and 9 pulmonary hypertension. Overall, 26 patients (84.0%) showed clinical improvement after ASD closure, including improved respiratory status (17/22; 77.3%), resumption of normal growth (15/24; 62.5%), and resolution of pulmonary hypertension (7/7; 100%, 2 patients unable to assess postoperatively). Two medically complicated patients died a few months after surgery unrelated to surgical complications. Four out of 8 ventilator-dependent patients were weaned from mechanical ventilation within 1 month after ASD closure. Closure of ASD did not improve those patients with highly advanced lung disease and/or medically complex conditions including underlying genetic abnormalities. Surgical complications were uncommon. Postoperative hospital stay was 4 to 298 days (median 8 days). The majority of our patients demonstrated significant clinical improvement after ASD closure. Early ASD closure is safe and beneficial for symptomatic infants and young children with associated underlying pulmonary abnormalities, especially bronchopulmonary dysplasia.

Mitral Valve Surgery in the First Year of Life.

Data are limited on outcomes associated with mitral valve surgery in infants. Prior studies report high mortality and increased risk for late cardiac failure particularly for those with mitral stenosis. We sought to evaluate outcomes in patients with mitral stenosis (MS) or regurgitation (MR) who had mitral valvuloplasty or replacement in the first year of life. A retrospective analysis of all patients in a single institution who underwent mitral valvuloplasty or replacement in their first year of life from 2004 to 2016 (n = 25), excluding patients with single ventricle pathology or those undergoing surgery for atrioventricular canal defect, was carried out. Median age and weight at surgery were 76.5 days (range 2-329) and 4.5 kg (range 3.0-10.1), respectively. The primary mitral pathology was MR in 16 and MS in 9 patients. Median follow-up among living patients was 4 years (range 106 days-12.3 years). Overall survival was 96% at 30 days and 87.8% at 1, 5, and 10 years. There were three early deaths (12%), all within 6 weeks of surgery. There were no late deaths. Three patients required valve replacement, 1 of which had a primary mitral valve replacement and died within 30 days of surgery. Re-intervention-free survival (surgical and catheter based) was 83.8%, 73.3%, and 48.9% at 1, 5, and 10 years per Kaplan-Meier estimates. There was no difference in re-intervention-free survival between patients with MR versus MS. No risk factors for death or re-intervention were identified. Mitral valvuloplasty and replacement can be performed in infants under 1 year of age with acceptable survival and need for re-intervention.

Feasibility of real-time cine cardiac magnetic resonance imaging to predict the presence of significant retrosternal adhesions prior to redo-sternotomy.

BACKGROUND: Injury to vital structures posterior to the sternum is a complication associated with redo sternotomy in congenital cardiac surgery. The goal of our study was a novel evaluation of real-time cine cardiovascular magnetic resonance (CMR) to predict the presence of significant retrosternal adhesions of cardiac and vascular structures prior to redo sternotomy in patients with congenital heart disease. METHODS: Twenty-three patients who had prior congenital heart surgery via median sternotomy had comprehensive CMR studies prior to redo sternotomy. The real time cine (RTC) sequence that was used is an ungated balanced steady-state free precession (bSSFP) sequence using SENSitivity Encoding for acceleration with real-time reconstruction. Spontaneously breathing patients were instructed to take deep breaths during the acquisition whilst increased tidal volumes were delivered to mechanically ventilated patients. All patients underwent redo cardiac surgery subsequently and the presence and severity of retrosternal adhesions were noted at the time of the redo sternotomies. RESULTS: Median age at the time of CMR and operation were 5.5 years (range, 0.2-18.4y) and 6.1 years (range, 0.3-18.8y) respectively. There were 15 males and 8 females in the study group. Preoperative retrosternal adhesions were identified on RTC in 13 patients and confirmed in 11 (85%) at the time of surgery. In only 2 patients, no adhesions were identified on CMR but were found to have significant retrosternal adhesions at surgery; false positive rate 15% (CI 0.4-29.6%), false negative rate 20% (CI 3.7-36.4%). The total classification error of the real time cine sequence was 17% (CI 1.7-32.4%) with an overall accuracy of 83% (CI 67.7-98.4%). Standard breath-hold cine images correlated poorly with surgical findings and did not increase the diagnostic yield. CONCLUSIONS: RTC imaging can predict the presence of significant retrosternal adhesions and thus help in risk assessment prior to redo sternotomy. These findings complement the surgical planning and potentially reduce surgical complications .

Variability in donor selection among pediatric heart transplant providers: Results from an international survey.

There is considerable variability in donor acceptance practices among adult heart transplant providers; however, pediatric data are lacking. The aim of this study was to assess donor acceptance practices among pediatric heart transplant professionals. The authors generated a survey to investigate clinicians' donor acceptance practices. This survey was distributed to all members of the ISHLT Pediatric Council in April 2018. A total of 130 providers responded from 17 different countries. There was a wide range of acceptable criteria for potential donors. These included optimal donor-to-recipient weight ratio (lower limit: 50%-150%, upper limit: 120%-350%), maximum donor age (25-75 years), and minimum acceptable left ventricular EF (30%-60%). Non-US centers demonstrated less restrictive donor selection criteria and were willing to accept older donors (50 vs 35 years, P < 0.001), greater size discrepancy (upper limit weight ratio 250% vs 200%, P = 0.009), and donors with a lower EF (45% vs 50%, P < 0.001). Recipient factors were most influential in the decision to accept marginal donors including recipients requiring ECMO support, ventilator support, and highly sensitized patients with a negative XM. However, programmatic factors impacted the decision to decline marginal donors including recent programmatic mortalities and concerns for programmatic restrictions from regulatory bodies. There is significant variation in donor acceptance practices among pediatric heart transplant professionals. Standardization of donor acceptance practices through the development of a consensus statement may help to improve donor utilization and reduce waitlist mortality.

Assessment of Growth 6 Years after the Norwood Procedure.

At 6 years of age, patients with hypoplastic left heart syndrome had mean age-adjusted z-scores for weight and height below the normative population, and body mass index was similar to the normative population. Males had the greatest increase in z-scores for body mass index. TRIAL REGISTRATION: ClinicalTrials.gov: NCT00115934.

Developmental screening in children with CHD: Ages and Stages Questionnaires.

OBJECTIVE: Standardised developmental screening tools are important for the evaluation and management of developmental disorders in children with CHD; however, psychometric properties and clinical utility of screening tools, such as the Ages & Stages Questionnaires, Third Edition (ASQ-3), have not been examined in the CHD population. We hypothesised that the ASQ-3 would be clinically useful for this population. Study design ASQ-3 developmental classifications for 163 children with CHD at 6, 12, 24, and/or 36 months of age were compared with those obtained from concurrent developmental testing with the Bayley Scales of Infant and Toddler Development, Third Edition. RESULTS: When ASQ-3 screening failure was defined as ⩾1 SD below the normative mean, specificity (⩾81.9%) and negative predictive value (⩾81.0%) were high across ASQ-3 areas. Sensitivity was high for gross motor skills (79.6%), increased with age for communication (35.7-100%), and generally decreased with age for problem solving (73.1-50.0%). When ASQ-3 screening failure was defined as ⩾2 SD below the normative mean, specificity (⩾93.6%) and positive predictive value (⩾74.5%) were generally high across ASQ-3 areas, but sensitivity was low (31.1%) to fair (62.8%). The ASQ-3 showed improved accuracy in predicting delays over clinical risk factors alone. CONCLUSIONS: The ASQ-3 appears to be a clinically useful tool for screening development in children with CHD, although its utility varied on the basis of developmental area and time point. Clinicians are encouraged to refer children scoring ⩾1 SD below the normative mean on any ASQ-3 area for formal developmental evaluation.

Emergent Interhospital Transport of Pediatric Patient With a Berlin Heart Device.

Ventricular assist devices (VADs) for the mechanical support of cardiac failure are being used more frequently in children of increasingly younger age. These children have significant and multiple medical comorbidities, and their length of hospital stay has been increasing. As this population of hospitalized VAD-supported children increases, so does the possibility of their need for interfacility transport for specialized diagnostic or therapeutic procedures. Reports on such transports are limited to 3 children who underwent scheduled elective transfers. We report our experience with a child with a Berlin Heart EXCOR left ventricular assist device (Berlin Heart, Berlin, Germany) who required emergent interfacility transport between our hospital and an affiliated institution.