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Introduction: Gaps in insurance coverage have been associated with diminished access to health care for children with chronic illness. Continuity of coverage is associated with improved long-term clinical outcomes among adolescent heart transplant (HT) recipients, but the relationship between insurance status and post-transplant outcomes has not been described across all pediatric HT recipients. Hypothesis: HT recipients with changes in insurance status will have worse outcomes as opposed to those with continuous coverage. Methods: We queried the United Network for Organ Sharing (UNOS) registry for patients age 0-18 receiving isolated HT or heart re-transplant between 2006 and 2019 and included them in the analysis if they survived at least one year post-transplant. Patients were categorized by insurance coverage at wait listing, HT, and one year follow-up. Cox proportional hazards models were used to characterize the association between coverage pattern and long-term patient survival. Results: Among the 3,247 patients in the analysis, insurance coverage patterns included continuous private (37%), continuous public (46%), gain of private (6%), and loss of private (11%). Patients who had continuous public insurance were more likely to be Black or Hispanic, adolescent, and require ICU care. In a multivariate Cox model, continuous public insurance was associated with increased mortality risk (HR = 1.46, P = 0.014), while loss of private insurance trended towards increased risk (HR = 1.14, P = 0.014). Figure 1 illustrates Kaplan-Meier conditional survival curves according to insurance trajectory. Multivariate sub-group analysis by Black race demonstrated no differences in outcomes by coverage pattern. Conclusions: In comparison to those with continuous private insurance, having continuous public insurance is associated with higher risk of adverse outcomes in pediatric HT recipients, but it does not account for disparate outcomes in Black patients.

Background: There are limited data describing the functional status (FS) of children after heart transplant (HT). We sought to describe the FS of children surviving at least 1 year after HT, to evaluate the impact of HT on FS, and to identify factors associated with abnormal FS post-HT. Methods: Organ Procurement and Transplantation Network data were used to identify all US children Results: A total of 1633 children met study criteria. At the 1-year assessment, 64% were “fully active/no limitations” (FSS=10), 21% had “minor limitations with strenuous activity” (FSS=9); and 15% scored ≤8. In comparison with listing FS, FS at 1 year post-HT increased in 91% and declined/remained unchanged in 9%. A stepwise regression procedure selected the following variables for association with abnormal FS at 1 year post-HT: ≥18 years of age (odds ratio [OR], 1.8; 95% confidence interval [CI], 1.2–2.7), black race (OR, 1.5; 95% CI, 1.1–2.0), support with ≥inotropes at HT (OR, 1.7; 95% CI, 1.2–2.5), hospitalization status at HT (OR, 1.5; 95% CI, 1.0–2.19), chronic steroid use at HT (OR, 1.5; 95% CI, 1.0–2.2), and treatment for early rejection (OR, 2.0; 95% CI, 1.5–2.7). Conclusion: Among US children who survive at least 1 year after HT, FS is excellent for the majority of patients. HT is associated with substantial improvement in FS for most children. Early rejection, older age, black race, chronic steroid use, hemodynamic support at HT, and being hospitalized at HT are associated with abnormal FS post-HT.

Background— Pediatric data on the impact of pre–heart transplantation (HTx) risk factors on early post-HTx outcomes remain inconclusive. Thus, among patients with previous congenital heart disease or cardiomyopathy, disease-specific risk models for graft loss were developed with the use pre-HTx recipient and donor characteristics. Methods and Results— Patients enrolled in the Pediatric Heart Transplant Study (PHTS) from 1996 to 2006 were stratified by pre-HTx diagnosis into cardiomyopathy and congenital heart disease cohorts. Logistic regression identified independent, pre-HTx risk factors. Risk models were constructed for 1-year post-HTx graft loss. Donor factors were added for model refinement. The models were validated with the use of patients transplanted from 2007 to 2009. Risk factors for graft loss were identified in patients with cardiomyopathy (n=896) and congenital heart disease (n=965). For cardiomyopathy, independent risk factors were earlier year of transplantation, nonwhite race, female sex, diagnosis other than dilated cardiomyopathy, higher blood urea nitrogen, and panel reactive antibody >10%. The recipient characteristic risk model had good accuracy in the validation cohort, with predicted versus actual survival of 97.5% versus 95.3% (C statistic, 0.73). For patients with congenital heart disease, independent risk factors were nonwhite race, history of Fontan, ventilator dependence, higher blood urea nitrogen, panel reactive antibody >10%, and lower body surface area. The risk model was less accurate, with 86.6% predicted versus 92.4% actual survival, in the validation cohort (C statistic, 0.63). Donor characteristics did not enhance model precision. Conclusions— Risk factors for 1-year post-HTx graft loss differ on the basis of pre-HTx cardiac diagnosis. Modeling effectively stratifies the risk of graft loss in patients with cardiomyopathy and may be an adjunctive tool in allocation policies and center performance metrics.

Background— Racial differences in long-term survival after heart transplant (HT) are well known. We sought to assess racial/ethnic differences in wait-list outcomes among patients listed for HT in the United States in the current era. Methods and Results— We compared wait-list and posttransplant in-hospital mortality among white, black, and Hispanic patients ≥18 years of age listed for their primary HT in the United States between July 2006 and September 2010. Of 10 377 patients analyzed, 71% were white, 21% were black, and 8% were Hispanic. Black and Hispanic patients were more likely to be listed with higher urgency (listing status 1A/1B) in comparison with white patients ( P Conclusions— Hispanic patients listed for HT in the United States appear to be at higher risk of dying on the wait-list or becoming too sick for a transplant in comparison with white patients. Black patients are not at higher risk of wait-list mortality, but they have higher early posttransplant mortality.

Background— Children listed for heart transplantation face the highest waiting list mortality in solid-organ transplantation medicine. We examined waiting list mortality since the pediatric heart allocation system was revised in 1999 to determine whether the revised allocation system is prioritizing patients optimally and to identify specific high-risk populations that may benefit from emerging pediatric cardiac assist devices. Methods and Results— We conducted a multicenter cohort study using the US Scientific Registry of Transplant Recipients. All children Conclusions— US waiting list mortality for pediatric heart transplantation remains unacceptably high in the current era. Specific high-risk subgroups can be identified that may benefit from emerging pediatric cardiac assist technologies. The current pediatric heart-allocation system captures medical urgency poorly. Further research is needed to define the optimal organ-allocation system for pediatric heart transplantation.

Background: The 30-day readmission rate is a well-established performance benchmark for adult heart failure programs. Little is known about the 30-day readmission rate for pediatric heart failure programs or the variability among centers. We sought to determine the 30-day readmission rate for children admitted with acute decompensated heart failure (ADHF) to support benchmarking efforts in pediatrics. Methods: The Pediatric Health Information System (PHIS) database was queried to identify all children ≤21 years of age admitted between January 2009 and March 2014 with a primary or secondary diagnosis of heart failure. All-cause 30-day readmission rates were calculated, as was mean length of stay. Results: Over the 5-year study period, a total of 39,150 hospitalizations at 47 children’s hospitals met the inclusion criteria. Overall, 56% were infants, 25% age 1-12 years, 9% adolescents, and 9% adults, 48% were female, 72% had congenital heart disease, 28% had cardiomyopathy/myocarditis. The mean hospital length of stay was 25 days (range 11,41 days; SD 7 days). In-hospital mortality was 8.4%. Overall 30-day readmission rate was 20% (range 10%, 30%; SD 5%) and varied considerably by center (Figure). Conclusion: A substantial portion of children (one in five) admitted to the hospital for heart failure is re-admitted within 30 days. There is considerable center-based variation in re-admission rates. The mean hospital length of stay exceeds 3 week and also varies considerably by center.

Background: Ventricular assist device (VAD)-related stroke is an important performance benchmark for adult VAD programs. No benchmarks are available for pediatric VAD programs where the stroke rate is higher. We sought to describe center variability in the stroke rate for the Berlin Heart EXCOR® Pediatric VAD, the most commonly used durable VAD in US children, to support pediatric VAD quality improvement efforts. Methods: All children age Results: Between 2007 and March 2014, a total of 320 children underwent implantation of a Berlin Heart EXCOR Pediatric VAD at 42 centers, of which 271 patients at 25 centers met the study inclusion criteria. The baseline characteristics of the cohort have been reported previously. Overall, there were a total of 64 neurological dysfunction episodes in 53 unique patients over 20,875 days of support, yielding an overall stroke incidence of 0.31 events per 100 days of support and a prevalence of 19%. Centers varied considerably (ten-fold) in their incidence rate of stroke ranging from 0 events to 1.7 events per 100 days of support (Figure). Among 13 centers with at least 10 total implants (totaling 205 patients over 14,405 support days), the overall stroke prevalence was 20% (range 6 to 46%) (Incidence rate 0.33 events per 100 days of support). Centers with ≥10 implants did not have fewer strokes than lower volume centers (P=0.30). Conclusion: The incidence of stroke among pediatric VAD centers using the Berlin Heart EXCOR Pediatric VAD was 0.31 events per day of support and varied considerably across pediatric centers. These data may be useful for developing performance benchmarks for neurological dysfunction in pediatric VAD support.

BACKGROUND: Use of pediatric mechanical circulatory support to decrease mortality has expanded dramatically. A national account of the use of FDA-approved devices in children is essential to understanding outcomes, refining patient selection, and improving quality of care. METHODS: PediMACs is an NIH-supported national registry for FDA-approved temporary and durable VAD usage in patients < 18 years of age. Between the launch in Sept 2012 and March 2014, 32 US hospitals enrolled patients. This first report of data from this registry analyzed pre-implant patient characteristics, survival using competing outcomes, and adverse events. RESULTS: One hundred and thirty pediatric pts underwent 153 VAD implantations. Etiology of heart disease included 87 (67%) pts with cardiomyopathy, 26 (20%) with congenital heart disease. Twenty pts transitioned from ECMO and 37 had prior cardiac surgery. Most pts were Intermacs level 1 (28%) and level 2 (55%) at implant, with 12% Intermacs level 3. There were 26 temporary devices. Of the durable devices, 65 (51%) were pulsatile and 62 (49%) continuous flow (CF) (compared to adult Intermacs implants: 4% pulsatile, 96% CF). Age at first implant included 22 pts (17%) < 1 yr of age, 23 (18%) 1-5 yrs, 26 (20%) 6-10 yrs and 59 (45%) 10-18 yrs. Patients were supported with LVAD alone in 99 (76%), BiVAD in 24 (18%) and total artificial heart in 2 (2%) and represented 484 months of follow-up. The 104 patients receiving primary durable devices had an actuarial survival of 90% at 6 mos. Competing risk analysis at 6 mos revealed 54% of pts transplanted, 37% alive on support, 8 % died and 1 % recovery. In the overall cohort, there were 8 early ( CONCLUSIONS: Pedimacs constitutes the largest single data repository with detailed information of pediatric pts implanted with all types of VADs. Initial data show a higher rate of use of pulsatile devices as compared to contemporaneous adult data. Survival rates are excellent despite varying patient characteristics and pump types. With further data collection, analysis of patient risk factors critical to improving outcomes will be possible.

Children with congenital and acquired heart disease are at risk for thrombotic events which, along with complications from thromboprophylaxis and antithrombotic therapy, are increasingly recognized as important contributors to morbidity and mortality in this heterogeneous group of patients.1 The prevalence and clinical relevance of thrombosis in these populations are influenced by developmental,2 acquired, and hemodynamic factors.3 Nevertheless, the pathophysiology, incidence, associated risk factors, and optimal strategies for prevention, detection/surveillance, and management of thrombosis in these populations have not been clearly defined by rigorous clinical investigations. The National Heart, Lung, and Blood Institute (NHLBI) convened a Working Group meeting on June 14 to 15, 2012 in Bethesda, Maryland, to explore the issues relevant to thrombosis in children with heart disease. The goals of the Working Group were to (1) review the current state of knowledge about thrombosis and incorporate additional knowledge derived from studies and guidelines relevant to adult populations, (2) identify gaps in the evidence base and prioritize opportunities for using basic and clinical research, and (3) identify common methodological issues and potential solutions to those issues for future studies in children. Given the broad topic, we purposely excluded discussion of cardiopulmonary bypass and extracorporeal membrane oxygenation, because these 2 topics are associated with unique issues and were thought to be beyond the focus of the Working Group. The Working Group consisted of experts in pediatric cardiology, hematology and neurology, adult cardiology and hematology, basic science of coagulation, imaging, clinical trial methodologies, quantitative analyses, and the dissemination and implementation of guidelines, along with representatives from the pharmaceutical industry and the Food and Drug Administration. This article represents the Working Group’s discussions on evidence gaps, challenges for this population, and recommendations for strategic research. There is insufficient evidence to guide the surveillance, prevention, and treatment of thrombosis in …

Background— Recent data suggest that Berlin Heart EXCOR Pediatric (EXCOR) ventricular assist device improves waiting list survival for pediatric heart transplant candidates. Little is known about their post-transplant outcomes. The aim of this analysis was to determine whether there was a difference in early survival for children bridged to transplant with EXCOR versus status 1A pediatric heart transplant patients not transplanted with ventricular assist device support. Methods and Results— Pediatric heart transplant patients (n=106) bridged to transplantation with EXCOR were compared with a similarly aged cohort (n=1021) within the Organ Procurement and Transplant Network (OPTN) database (both cohorts from May 2007 to December 2010). In the EXCOR group, 12-month post-transplant survival (88.7%) was similar to OPTN patients listed status 1A who were not on ventricular assist device support at transplant (89.3%; P =0.85) and significantly better than 12-month survival in OPTN patients on extracorporeal membrane oxygenation at transplant (60.3%; P P =0.005) higher cause of 12-month post-transplant mortality in the EXCOR compared with the OPTN group. Death after transplant was also higher in EXCOR patients with congenital heart disease compared with those with cardiomyopathy (26.1% versus 7.2%; P =0.02). Post-transplant survival was similar in EXCOR patients with ≥1 serious adverse event during ventricular assist device support as those without an event during support. Conclusions— The 12-month post-transplant survival with EXCOR is comparable with overall pediatric heart transplant survival and superior to survival after extracorporeal membrane oxygenation. Neither adverse events during support nor factors associated with mortality during support influence post-transplant survival. Rejection was a significantly greater cause of post-transplant mortality in EXCOR than in OPTN patients.

Background— Recent data suggest that the Berlin Heart EXCOR Pediatric ventricular assist device is superior to extracorporeal membrane oxygenation for bridge to heart transplantation. Published data are limited to 1 in 4 children who received the device as part of the US clinical trial. We analyzed outcomes for all US children who received the EXCOR to characterize device outcomes in an unselected cohort and to identify risk factors for mortality to facilitate patient selection. Methods and Results— This multicenter, prospective cohort study involved all children implanted with the Berlin Heart EXCOR Pediatric ventricular assist device at 47 centers from May 2007 through December 2010. Multiphase nonproportional hazards modeling was used to identify risk factors for early ( Conclusions— Use of the Berlin Heart EXCOR has risen dramatically over the past decade. The EXCOR has emerged as a new treatment standard in the United States for pediatric bridge to transplantation. Three-quarters of children survived to transplantation or recovery; an important fraction experienced neurological dysfunction. Smaller patient size, renal dysfunction, hepatic dysfunction, and biventricular assist device use were associated with mortality, whereas extracorporeal membrane oxygenation before implantation and congenital heart disease were not.

Background— Extracorporeal membrane oxygenation (ECMO) has served for >2 decades as the standard of care for US children requiring mechanical support as a bridge to heart transplantation. Objective data on the safety and efficacy of ECMO for this indication are limited. We describe the outcomes of ECMO as a bridge to heart transplantation to serve as performance benchmarks for emerging miniaturized assist devices intended to replace ECMO. Methods and Results— Data from the Extracorporeal Life Support Organization Registry and the Organ Procurement Transplant Network database were merged to identify children supported with ECMO and listed for heart transplantation from 1994 to 2009. Independent predictors of wait-list and posttransplantation in-hospital mortality were identified. Objective performance goals for ECMO were developed. Of 773 children, the median age was 6 months (interquartile range, 1 to 44 months); 28% had cardiomyopathy; and in 38%, a bridge to transplantation was intended at ECMO initiation. Overall, 45% of subjects reached transplantation, although one third of those transplanted died before discharge; overall survival to hospital discharge was 47%. Wait-list mortality was independently associated with congenital heart disease, cardiopulmonary resuscitation before ECMO, and renal dysfunction. Posttransplantation mortality was associated with congenital heart disease, renal dysfunction, ECMO duration of >14 days, and initial ECMO indication as a bridge to recovery. In the objective performance goal cohort (n=485), patients with cardiomyopathy had the highest survival to hospital discharge (63%), followed by patients with myocarditis (59%), 2-ventricle congenital heart disease (44%) and 1-ventricle congenital heart disease (33%). Conclusion— Although ECMO is effective for short-term circulatory support, it is not reliable for the long-term circulatory support necessary for children awaiting heart transplantation. Fewer than half of patients bridged with ECMO survive to hospital discharge. More effective modalities for chronic circulatory support in children are urgently needed.

BACKGROUND UNOS (United Network for Organ Sharing) data suggest that of all pediatric transplant candidates, infants awaiting heart transplantation face the highest waitlist mortality. We sought to determine the mortality rate for waitlisted infants in the current era and to identify the risk factors associated with waitlist mortality. METHODS In this multi-center cohort study using the US Scientific Registry of Transplant Recipients, we analyzed all infants RESULTS In this six year period, 1133 patients less than 12 months of age were identified with a median age of 1 month (IQR 0 to 5 mo) and median weight of 4.0 kg (IQR 3.2 to 5.6 kg). 516 (45%) were female, 438 (39%) were non-white, 724 (64%) had congenital heart disease (CHD) and 910 (80%) were listed status 1A. Overall, 250 (22%) died while waiting including 179 (72%) listed at age CONCLUSION Mortality of infants waiting for heart transplantation remains high with a 22% waitlist mortality in the current era. ECMO support, ventilator support, CHD, age less than 3 months, and non-white race were independently associated with waitlist mortality whereas listing status was not. Measures to improve donor availability for this age group and refinements in organ allocation to lower waitlist mortality are urgently needed.