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The Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs), supported by the Society of Thoracic Surgeons, provides detailed information on pediatric patients supported with ventricular assist devices (VADs).From September 19, 2012, to December 31, 2021, there were 1,355 devices in 1,109 patients (19 years) from 42 North American Hospitals.Cardiomyopathy was the most common underlying etiology (58%), followed by congenital heart disease (CHD) (26%) and myocarditis (9%). Regarding device type, implantable continuous (IC) VADs were most common at 40%, followed by paracorporeal pulsatile (PP) (28%), and paracorporeal continuous (PC) (27%). Baseline demographics differed, with the PC cohort being younger, smaller, more complex (ie. CHD), and sicker at implant (P.0001). At six months post-VAD implantation, a favorable outcome (transplant, recovery, or alive on device) was achieved in 84% of patients, which was greatest among those on IC VADs (92%) and least for PC VADs (69%). Adverse events (AEs) were not uncommon, with non-gastrointestinal bleeding (incidence of 14%) and neurological dysfunction (11% [Stroke 4%]), within 2 weeks post implant being the most prevalent. Stroke and bleeding had negative impacts on overall survival (p=0.002 and p0.001, respectively).This Sixth Pedimacs Report demonstrates the continued evolution of the pediatric field. The complexity of cardiac physiologies and anatomical constraint mandates the need for multiple types of devices utilized (PC, PP, IC). Detailed analyses of each device type in this report provide valuable information to further advance the care of this challenging and vulnerable population.

Individuals with Fontan circulation are at risk of late mortality from both cardiac and noncardiac causes. Despite the known risk of mortality, referral indications for advanced heart failure care vary between centers, and many individuals die from Fontan circulation-related complications either after late consideration for advanced heart failure therapies or having never seen a heart failure specialist. There is a critical need for guidelines to direct appropriately timed referral for advanced heart failure consultation. The Advanced Cardiac Therapies Improving Outcomes Network (ACTION) Fontan Committee has developed recommended thresholds for advanced heart failure referral to guide primary cardiologists. These recommendations are divided into 4 categories of clinical Fontan circulatory dysfunction including (1) cardiac/systemic ventricular dysfunction, (2) Fontan pathway dysfunction, (3) lymphatic dysfunction, and (4) extracardiac dysfunction.

Data on ventricular assist device (VAD) outcomes in infants with stage 1 single ventricle (SV) palliation are limited. We examined the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) registry for outcomes of pre/poststage 1 SV patients undergoing VAD implantation between March 2018 and October 2020. Data are collected from 32 centers and major adverse events undergo central adjudication. During the study period, 30 stage 1 SV patients underwent VAD implant with median age of 0.9 months (range 0.1-25) and weight 3.7 kg (2.4-17). Preimplant illness severity was high: ventilator support (90%), ECMO (30%), prior cerebral vascular accident (CVA, 23%), and dialysis (13%). Devices used included paracorporeal pulsatile (50%), paracorporeal continuous-flow (37%), and multiple devices (13%). Median support duration was 56 days (range 3-246). A positive clinical outcome (transplanted or weaned) was attained in 63% (63% transplanted, 37% mortality, 0% weaned). VAD adverse events included: major infection (43%), neurologic dysfunction (any = 30%; CVA = 20%), major bleeding (17%), renal dysfunction (13%), and device malfunction (3%). In conclusion, stage 1 SV patients undergoing VAD support have high preimplant illness severity and complexity, as well as significant morbidity and mortality postimplant. A variety of devices and strategies are employed by centers to support this challenging population.

BACKGROUND The size of the Fontan population with end-stage heart failure is growing. In this population, heart transplantation has been the only option. This study sought to investigate the efficacy of ventricular assist device (VAD) support in Fontan patients. METHODS We conducted a retrospective study of Fontan patients in the Advanced Cardiac Therapies Improving Outcomes Network. We evaluated patient characteristics, and the clinical and physiologic outcomes after VAD implantation. RESULTS We identified 45 Fontan patients implanted with VAD. The average age of patients was 10 years (interquartile range: 4.5–18) and 30% were female. The majority had a morphologic right ventricle (69%), moderate or greater ventricular dysfunction (83%), and moderate or greater atrioventricular valve regurgitation (65%). The majority of implants were as a bridge to transplantation (76%), and the majority of patients were Interagency Registry for Mechanically Assisted Circulatory Support Profile 2 (56%). The most commonly employed device was the Medtronic HeartWare HVAD (56%). A total of 13 patients were discharged on device support, and 67% of patients experienced adverse events, the most common of which were neurologic (25%). At 1 year after device implantation, the rate of transplantation was 69.5%, 9.2% of patients continued to be VAD supported, and 21.3% of patients had died. Hemodynamically, VAD was effective in decreasing both Fontan and ventricular end-diastolic pressures in some individuals. CONCLUSIONS VAD is effective in supporting patients with end-stage Fontan failure awaiting heart transplantation. Future research should focus on identifying clinical and physiologic characteristics predictive of a favorable response to VAD support.

BACKGROUND The Berlin Heart EXCOR Pediatric (EXCOR) ventricular assist device (VAD) was introduced in North America nearly 2 decades ago. The EXCOR was approved under Humanitarian Device Exemption status in 2011 and received post-market approval (PMA) in 2017 from Food and Drug Administration. Since the initial approval, the field of pediatric mechanical circulatory support has changed, specifically with regard to available devices, anticoagulation strategies, and the types of patients supported. This report summarizes the outcomes of patients supported with EXCOR from the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) registry. These data were part of the PMA surveillance study (PSS) required by the Food and Drug Administration. METHODS ACTION is a learning collaborative of over 40 pediatric heart failure programs worldwide, which collects data for all VAD implantations as one of its initiatives. All patients in North America with EXCOR implants reported to ACTION from 2018 to 2020 ( n = 72) who had met an outcome were included in the EXCOR PSS group. This was compared with a historical, previously reported Berlin Heart EXCOR study group (Berlin Heart study [BHS] group, n = 320, 2007‒2014). RESULTS Patients in the PSS group were younger, were smaller in weight/body surface area, were more likely to have congenital heart disease , and were less likely to receive a bi-VAD than those in the BHS group. Patients in the PSS group were less likely to be in Interagency Registry for Mechanically Assisted Circulatory Support Profile 1 and were supported for a longer duration. The primary anticoagulation therapy for 92% of patients in the PSS group was bivalirudin . Success, defined as being transplanted, being weaned for recovery, or being alive on a device at 180 days after implantation, was 86% in the PSS group compared with 76% in the BHS group. Incidence of stroke was reduced by 44% and the frequency of pump exchange by 40% in the PSS group compared with those in the BHS group. Similarly, all other adverse events , including major bleeding, were reduced in the PSS group. CONCLUSIONS The PSS data, collected through ACTION, highlight the improvement in outcomes for patients supported with EXCOR compared with the outcomes in a historical cohort. These findings may be the result of changes in patient care practices over time and collaborative learning.

We sought to describe QOL in children with VAD and to identify factors associated with impaired QOL.There were 82 children (6-19 years) in the Pediatric Interagency Registry for Mechanical Circulatory Support who completed the PedsQL +/- a VAD-specific QOL assessment pre-VAD implant (n = 18), 3 months post-VAD (n = 63), and/or 6 months post-VAD (n = 38). Significantly impaired QOL is a score1 SD below norms.Study patients were 59% male, 67% Caucasian, with cardiomyopathy diagnosis in 82%, and median age at implant of 14 y (IQR 11-17). PedsQL scores were lower than norms for physical (p .0001) and psychosocial (p .01) QOL in pre- and post-VAD groups. Compared to chronic health condition and complex or severe heart disease groups, PedsQL scores were lower for physical and psychosocial QOL in the pre-VAD group (p .0001); however, psychosocial QOL was not significantly different in post-VAD groups. Psychosocial QOL was impaired in 67%, 40%, and 24% in pre-VAD, 3-month, and 6-month post-VAD groups, respectively. Total and psychosocial QOL scores were significantly higher in the 3-month and 6-month post-VAD group than pre-VAD (all p ≤ .02). VAD patients were most bothered by their inability to participate in usual play activities. Impaired QOL 3 months post-VAD was associated with inotropic support2 weeks/ongoing post-VAD (p = .04).Physical QOL is significantly impaired in most children pre- and post-VAD. However, psychosocial QOL is not significantly impaired in most children post-VAD suggesting VAD implantation may improve psychosocial QOL in children.

We sought to characterize strokes in children with ventricular assist devices. Of 407 patients in the ACTION registry (4/1/18-5/3/2021), 45 (11%) experienced 52 strokes (45 ischemic and 7 hemorrhagic). Median time to stroke was 23.5 days and 19/52 (37%) occurred ≤ 10 days. Stroke rate was 0.09 and 0.63 strokes per patient-year for implantable continuous and paracorporeal devices, respectively. Patients with stroke were younger, more likely to have congenital heart disease and have been on extracorporeal membrane oxygenation at time of VAD. Based on these data, ACTION is now focused on decreasing strokes in these higher-risk patients with particular attention to the peri-implant period.

BACKGROUND The HeartMate 3 ventricular assist device (VAD) is a newer centrifugal continuous-flow VAD used for bridge-to-transplant and destination therapy in adults. However, there is limited experience regarding its use in children and adults with complex congenital heart disease (CHD). METHODS The Advanced Cardiac Therapies Improving Outcomes Network (ACTION) is a multicenter learning network comprised of pediatric hospitals implanting VADs in children and adults with complex CHD. We examined the outcomes of patients undergoing HeartMate 3 implantation at an ACTION center between December 2017 and September 2019. RESULTS The HeartMate 3 was implanted in 35 patients at 9 ACTION centers, with a median age of 15.7 (8.8–47.3) years, median weight of 65.7 (19.1–114.1) kg, and median body surface area (BSA) of 1.74 (0.78–2.36) m2. Of the cohort, 14 patients (40%) weighed CONCLUSIONS Use of the HeartMate 3 in ACTION centers was associated with a low incidence of mortality and adverse events. Patients as small as 19 kg (BSA 0.78 m2) were successfully implanted and supported, indicating that this device may be appropriate for older children and small adults.

The number of potential pediatric heart transplant recipients continues to exceed the number of donors, and consequently the waitlist mortality remains significant. Despite this, around 40% of all donated organs are not used and are discarded. This document (62 authors from 53 institutions in 17 countries) evaluates factors responsible for discarding donor hearts and makes recommendations regarding donor heart acceptance. The aim of this statement is to ensure that no usable donor heart is discarded, waitlist mortality is reduced, and post-transplant survival is not adversely impacted.

Ventricular assist devices (VADs) have become a mainstay of advanced heart failure therapy in pediatrics, with outcomes in adolescents that parallel the excellent outcomes in adults. Unfortunately, outcomes for younger children and infants remain suboptimal. This review discusses the patient and device specifics that contribute to this discrepant outcome, highlight current treatment strategies, and suggest areas for future improvement. The field of pediatric mechanical circulatory support (MCS) continues to be driven by advancements in patient selection, timing of VAD implantation, and patient management strategies particularly with regard to anticoagulation. The use of direct thrombin inhibitors, such as bivalirudin, and weight-based antiplatelet therapy has shown promise in reducing the incidence of bleeding and stroke. While the number of devices remains limited, providers continue to leverage current technology and novel cannulation strategies to support smaller and more complex patients. Despite the challenges of supporting infants and small children, it remains a hopeful time for the field of pediatric MCS. While individual centers may only care for small number of VAD patients, the ACTION learning network has sparked collaboration across the field broadly with rapid dissemination of best practices to all centers. This collaboration has led to many advances and holds promise for driving future advancements.

The use of ventricular assist device (VAD) in children has increased, but the decision of left VAD (LVAD) vs biventricular support remains a challenge. Children who undergo LVAD placement are at risk for right ventricular failure (RHF), but the incidence has not been described.Analysis was performed for patients18 years old who underwent durable LVAD placement within the Pedimacs registry (September 19, 2012-February 28, 2017), excluding single ventricle morphology and temporary devices. RHF was defined as the need for right ventriculalr assist device (RVAD) or prolonged inotrope use between 1 week to 1 month and 1 to 3 months. End-points included death, heart transplant (HT), and recovery.A total of 272 durable LVAD were placed of which 37 died on device over 24 month follow-up, primarily from multiorgan failure and neurologic dysfunction. RVAD occurred in 12 children at median 8.5 days, with 9 undergoing HT and 3 dying on device. In patients with only LVAD, RHF was present in 111/207 (55%) between 1 week to 1 month and 28/116 (25%) between 1 and 3 months. Younger age, smaller weight, Intermacs profile 1, chemical paralysis, and pulsatile flow VAD were associated with RHF. RHF was associated with increased risk of death on device at both1 month (hazard ratio 3.2, 95% CI 1.4-7.7, p = 0.007) and3 month (hazard ratio 6.9, 95% CI 2-23.1, p = 0.002).In children, RHF is common after durable LVAD implantation, but subsequent RVAD is relatively rare. RHF in children, as indicated by prolonged inotrope support, was associated with an increased risk of death on the device. Whether early RVAD support and higher waitlist status may improve the outcome remains unknown.

Introduction: Risk estimation for waitlist and 1-year post-transplant mortality utilized by SRTR for pediatric heart transplantation dichotomizes patients based upon presence or absence of congenital heart disease (CHD). Hypothesis: Current SRTR risk estimations can be improved by adding further detail in categorization of CHD. Methods: A retrospective analysis of the Pediatric Heart Transplant Society (PHTS) database of pediatric ( Results: The WaitDeath model included 5,790 patients in which 722 of which died. In the SRTR WaitDeath model, CHD had a HR of 1.8 (95%CI of 1.4-2.3) compared to cardiomyopathy patients. Where as in SRTR+ model, patients with different types of CHD had HR’s that ranged from 1.5 to 2.1 (Table 1). The TxDeath model included 4,185 patients in which 304 which died. (Table 1) In the SRTR model of TxDeath, patients with CHD had a HR of 3.9 (2.9-5.1) compared to cardiomyopathy patients, whereas in the SRTR+ model patients with different types of CHD had HRs that ranged from 2.4-5.3 (Table 1). Conclusions: Inclusion of granular CHD data may improve waitlist and post-transplant mortality risk estimation in pediatric heart transplant patients, allowing for more refinement in allocation policies and understanding of difference in post-transplant outcomes across sites. This data supports further refinement of current SRTR risk adjustment models.

BACKGROUND We aimed to describe the incidence, risk factors, and clinical outcomes of pericardial effusions within 6 months after pediatric heart transplantation (HT). METHODS A single-center retrospective cohort study was performed on all pediatric HT recipients from 2004 to 2018. Logistic regression was used to identify factors associated with pericardial effusions post-HT, and survival was compared using log-rank test. RESULTS During the study period, 97 HTs were performed in 93 patients. Fifty patients (52%) had a ≥small pericardial effusion within 6 months, 16 of which were, or became, ≥moderate in size. Pericardial drain was placed in 8 patients. In univariate analysis, larger recipient body surface area (p = .01) and non-congenital heart disease (p = .002) were associated with pericardial effusion development. Donor/recipient size ratios, post-HT hemodynamics, and rejection did not correlate with pericardial effusion development. In multivariable analysis, non-congenital heart disease (adjusted odds ratio 3.3, p = .01) remained independently associated with development of pericardial effusion. There were no significant differences in post-HT survival between patients with and without ≥small (p = .68) or ≥moderate pericardial effusions (p = .40). CONCLUSIONS Pericardial effusions are common after pediatric HT. Patients with cardiomyopathy, or non-congenital heart disease, were at higher risk for post-HT pericardial effusions. Pericardial effusions increased morbidity but had no effect on mortality in our cohort. The risk factors identified may be used for anticipatory guidance in pediatric HT.

The Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) provides detailed information on pediatric patients supported with ventricular assist devices (VADs).From September 19, 2012, to December 31, 2020, 1229 devices in 1011 patients were reported to the registry from 47 North American Hospitals in patients aged younger than 19 years.Cardiomyopathy was the most common underlying etiology (58%), followed by congenital heart disease (CHD; 25%) and myocarditis (10%). The most common devices implanted were implantable continuous (IC; 419 [41%]), followed by paracorporeal pulsatile (PP; 269 [27%]), paracorporeal continuous (PC; 263 [26%]), and percutaneous (53 [5%]). Overall, at 6 months after VAD implantation, 83% had a positive outcome (transplant, explant, or alive on device). The freedom from stroke at 3 months was highest in IC VADs (93%), compared with PP VADs (84%) and with PC VADs (75%). There were differences in survival by device type, with patients on IC VADs having the best overall survival and those on PC having the lowest overall survival, though the patient populations being supported by each VAD type differed significantly from each other.This Fifth Pedimacs Report demonstrates the continued robust growth of VADs in the pediatric community, now with more than 1000 patients reported to the registry. The multiple available device types (PC, PP, IC) serve different populations with different pre-VAD risk profiles, which may account for differences in survival and adverse events between device types.

Anemia is a predictor of morbidity and mortality in both pediatric and adult patients with heart failure. This risk is increased in patients who require ventricular assist device (VAD) placement. The most common mechanism suggested for why these patients develop anemia is chronic inflammation caused by the immune system reacting to the VAD components. The inflammatory response that occurs can suppress erythropoiesis by inhibiting production of erythropoietin. Studies have demonstrated that anemic VAD patients have lower-than-expected erythropoietin levels, which leads to the consideration of erythropoiesis-stimulating agents (ESAs) in this population. Therapy with ESAs can increase hemoglobin and hematocrit levels, thereby decreasing the need for transfusions, subsequently reducing the risk of anti–human leukocyte antigen antibody development. Concerns that ESAs may increase the risk of thrombotic complications in a population already plagued with physiologic disturbances due to the VAD device remain a main barrier in routine use of these medications. The goal of this case series is to discuss a single center's experience with epoetin alfa in pediatric VAD patients at an academic children's hospital. A total of 4 patients were included with no evidence of adverse effects during a total of 120 patient-days of epoetin therapy. One patient was able to discontinue ESA therapy secondary to robust improvement in cell line counts at the time of discharge, while the other 3 patients received heart transplant prior to the discontinuation of ESA therapy. An increase in hematocrit of 1% to 5.5% was seen from epoetin initiation to discontinuation.

The success of ventricular assist devices (VADs) in the treatment of end-stage heart failure in the adult population has led to industrial innovation in VAD design, focusing on miniaturization and the reduction of complications. A byproduct of these innovations was that newer generation devices could have clinical applications in the pediatric population. Over the last decade, VAD usage in the pediatric population has increased dramatically, and the newer generation continuous flow (CF) devices have begun to supplant the older, pulsatile flow (PF) devices, formerly the sole option for ventricular assist in the pediatric population. However, despite the increase in VAD implants in the pediatric population, patient numbers remain low, and the need to share data between pediatric VAD centers has become that much more important for the continued growth of VAD programs worldwide. The creation of pediatric VAD registries, such as the Pediatric Registry for Mechanical Circulatory Support (PediMACS), the European Registry for Patients with Mechanical Circulatory Support (EUROMACS) and the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) has enabled the collection of aggregate data from VAD centers worldwide, and provides a valuable resource for clinicians and programs, as more and more pediatric heart failure patients are considered candidates for VAD therapy.

We sought to assess acute hemodynamic changes after implementation of negative extrathoracic pressure (NEP) in spontaneously breathing ambulatory Fontan patients with symptomatic heart failure. We hypothesized that application of NEP would result in an acute decrease in pulmonary artery pressure. Ten patients with clinical evidence of Fontan failure underwent baseline hemodynamic catheterization while breathing spontaneously. Hemodynamic measurements were then repeated after 30 min of continuous NEP. After 30 min of continuous NEP, 4/10 patients had a decrease in their Fontan pressure by 2 mmHg and one patient had a decrease by 1 mmHg. There were three patients that had an increase in Fontan pressure by 2 mmHg. In 7/10 patients, indexed pulmonary vascular resistance decreased by an average of 31%. In symptomatic Fontan patients with a favorable hemodynamic response to NEP during catheterization, potential benefit of longer-term NEP to improve clinical status should be explored.

Despite increasing utilization of continuous-flow pediatric ventricular assist devices (VAD) in children, data on exercise testing and cardiac rehabilitation (CR) are unknown. We described variation in CR practices and identified barriers to exercise testing and CR. A survey was performed through the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) representing pediatric VAD centers across North America. Descriptive statistics were performed. A multidisciplinary cohort of 52 respondents from 28 pediatric VAD centers responded. Although 38% reported performing exercise testing, most (65%) used 6 minute walk tests rather than formal cycle or treadmill exercise testing. While all respondents refer to physical therapy during the initial inpatient stay for VAD placement, only 52% refer to a CR program. When performed, CR was performed at an ACTION center (84%), a local specialized center (21%), or a home-based CR program (26%). Commonly cited barriers to either CR or exercise testing were inadequate resources, inadequate implementation logistics knowledge, concerns about safety, inability of patients to travel to a CR facility, and concern about utility of exercise testing or CR. Over 90% of centers were interested in implementing a standardized pediatric VAD CR program. Utilization of exercise testing and CR after VAD placement is variable. Despite perceived barriers, most pediatric VAD centers are interested in implementing a standardized CR program for recipients. In response to this interest, we plan to implement a standardized CR protocol to all ACTION pediatric VAD centers in an effort to improve pretransplant waitlist rehabilitation and post-transplant outcomes.

Right heart failure (RHF) is a vexing problem in children after left ventricular assist device (LVAD) implantation that can negatively impact transplant candidacy and survival. Anticipation, prevention, early identification and appropriate medical and device management of RHF are important to successful LVAD outcomes. However, there is limited pediatric evidence to guide practice. This pediatric-focused review summarizes the relevant literature and describes the harmonized approach to RHF from the Advanced Cardiac Therapies Improving Outcomes Network (ACTION). This review seeks to improve RHF outcomes through the sharing of best practices and experience across the pediatric VAD community.

Background In a stable, inotrope-dependent pediatric patient with dilated cardiomyopathy, we evaluated the cost-effectiveness of continuous-flow VAD implantation compared to a watchful waiting approach using chronic inotropic therapy. Methods We used a state-transition model to estimate the costs and outcomes of 14-year-old (INTERMACS profile 3) patients receiving either VAD or watchful waiting. We measured benefits in terms of lifetime QALYs gained. Model inputs were taken from the literature. We calculated the ICER, or the cost per additional QALY gained, of VADs and performed multiple sensitivity analyses to test how our assumptions influenced the results. Results Compared to watchful waiting, VADs produce 0.97 more QALYs for an additional $156 639, leading to an ICER of $162 123 per QALY gained from a healthcare perspective. VADs have 17% chance of being cost-effective given a cost-effectiveness threshold of $100 000 per QALY gained. Sensitivity analyses suggest that VADs can be cost-effective if the costs of implantation decrease or if hospitalization costs or mortality among watchful waiting patients is higher. Conclusions As a bridge to transplant, VADs provide a health benefit to children who develop stable, inotrope-dependent heart failure, but immediate implantation is not yet a cost-effective strategy compared to watchful waiting based on commonly used cost-effectiveness thresholds. Early VAD support can be cost-effective in sicker patients and if device implantation is cheaper. In complex conditions such as pediatric heart failure, cost-effectiveness should be just one of many factors that inform clinical decision-making.

Purpose Ventricular assist device (VAD) use in patients with congenital heart disease is increasing. Data on VAD outcomes in infants/children with stage 1 single ventricle (SV) are limited. Our study aims to characterize the outcomes of this population using multi-center registry data. Methods We examined the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) registry for outcomes of stage 1 SV patients undergoing VAD implantation between March 2018 - June 2020. ACTION data are collected from 30 centers, and major adverse events undergo central adjudication using pre-specified event definitions. Clinical characteristics, major adverse events, and survival are reported. Results During the study period, 27 stage 1 SV patients underwent VAD implant with median age of 1 month (range 0.1-44), weight 3.7kg (2.4-17), and BSA 0.24 m2 (0.18-0.65). Pre-implant comorbidities were frequent: ventilator support (85%), ECMO (37%), history of cerebral vascular accident (CVA, 30%), and pre-implant dialysis (18.5%). Device strategy included paracorporeal continuous-flow VAD in 11 patients, paracorporeal pulsatile VAD (12), and multiple devices (4). Concomitant surgical procedure was performed in 4 patients. Median support duration was 56 days (2-271). A positive clinical outcome, defined as transplant, weaned, or alive on device was obtained in 59%, with 12 patients transplanted while 11 died on device (Fig 1). Adverse events on VAD support included neurologic dysfunction (18.5%, with n=2 CVA), bleeding (22%), infection (48%), renal dysfunction (15%), and device malfunction (4%). Conclusion Stage 1 SV patients undergoing VAD support have frequent and severe pre-implant comorbidities, which likely contributed to the significant morbidity and mortality post-implant. A variety of device types and surgical strategies are employed by centers to support this challenging physiology.

Purpose ACTION (Advanced Cardiac Therapies Improving Outcomes Network) is a multi-faceted quality improvement (QI) and research network with the goal of improving pediatric and congenital heart disease VAD and heart failure (HF) outcomes. This report describes ACTION initiatives. Methods ACTION includes QI, educational activities, and a data registry. Stakeholders include providers, patients and families, and industry partners. The core values of ACTION include collaboration and transparency. ACTION started in 2017, with data collection beginning in April 2018. This report utilizes ACTION data and describes selected completed and ongoing initiatives. Results The ACTION registry report is shared with sites as an interactive dashboard (refreshed monthly). A comprehensive data quality strategy includes data queries and adjudication of critical events. There are 50 US/Canadian sites in the network and 34 of the sites contribute data. There are additional international sites that participate in all activities except patient-level data collection. The registry includes 388 patients implanted with 511 devices with 51,411 device days. DCM (203) and CHD (143) are the major underlying diagnoses. EXCOR (148), HVAD (97), and HM3 (70), are the most common end devices. Survival is 91% (including ongoing support, transplant and wean). AE rates are: stroke 11%, bleeding 21%, and infection 26%. The dashboard allows intuitive examination of subgroups, e.g., stroke/bleeding rate in patients less than 10 kg/on a specific device. Initial QI projects include an initiative (ABC of stroke), that used a bundle of interventions to reduce stroke during VAD support; a discharge planning project; a telehealth support project; and a harmonization initiative to reduce burden of protocol development for sites. Additionally, a patient-facing education website about VAD/HF care is being deployed and a HF project is collecting baseline data for acute decompensated HF. ACTION data have been used to support regulatory (FDA) approval of selected devices. Conclusion ACTION, a collaborative, multi-stakeholder network, has undertaken numerous approaches to improve outcomes for children with HF and VADs. In keeping with the values of transparency and collaboration, the ACTION annual reports will be made available in their entirety in a patient-facing version, via the ACTION website.

Background The Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs), originally a National Institutes of Health–sponsored U.S. database, provides a platform to understand the population of children supported with ventricular assist devices (VADs) during this time of increasing numbers, new devices, expanding indications, and improved outcomes. Methods Between September 19, 2012, and December 31, 2019, 44 hospitals implanted 1031 devices in 856 patients under 19 years of age. Results Overall, diagnosis was cardiomyopathy in 497 (58%) patients, congenital heart disease (CHD) in 216 (25%), myocarditis in 85 (10%), and other in 58 (7%). Positive outcome (alive on device or bridge to transplantation and recovery) occurred in 82% at 6 months. The patient cohort for implantable continuous (IC) pumps (n = 365) (age 13.2 ± 3.9 years, 18% Interagency Registry for Mechanically Assisted Circulatory Support [Intermacs] profile 1, 23% intubated at implantation, 16% with CHD) was significantly different from the paracorporeal continuous (PC) pump cohort (n = 212) (age 3.6 ± 4.9 years, 46% Intermacs profile 1, 81% intubated, 42% CHD) and the paracorporeal pulsatile (PP) pump cohort (n = 230) (age 2.7 ± 3.5 years, 31% Intermacs profile 1, 76% intubated, 26% CHD). Consistent with their cohort composition, positive outcomes at 6 months based on device type were the following: IC, 92%; PC, 68%; and PP, 81%. The incidence of cerebrovascular accidents in the IC, PC, and PP cohorts was 7%, 14%, and 15%, respectively. Conclusions IC VADs, the most common VAD type placed in children, are associated with improved outcomes compared with PP and PC devices, though PP and PC devices are limited to supporting our most challenging patients. Noteworthy, the incidence of cerebrovascular accidents for pediatric VADs has significantly decreased and is now 11% overall. This report demonstrates again that although often attributed to age, size, or device type, much of the burden in mortality and adverse events is correlated to the patient’s overall state at VAD implantation.

Improving the outcomes of pediatric patients with congenital heart disease with end-stage heart failure depends on the collaboration of all stakeholders; this includes providers, patients and families, and industry representatives. Because of the rarity of this condition and the heterogeneity of heart failure etiologies that occur at pediatric centers, learnings must be shared between institutions and all disciplines to move the field forward. To foster collaboration, excel discovery, and bring data to the bedside, a new, collaborative quality improvement science network-ACTION (Advanced Cardiac Therapies Improving Outcomes Network)-was developed to meet the needs of the field. Existing gaps in care and the methods of improvement that will be used are described, along with the mission and vision, utility of real-world data for regulatory purposes, and the organizational structure of ACTION is described.

Background Patients listed for heart transplant after their 18 th birthday purportedly wait longer to receive a donor heart compared to patients listed before their 18 th birthday. It is unclear whether there is an actual difference in wait times and whether any difference in wait time is associated with lower likelihood of transplant and/or higher risk of mortality. Methods Organ procurement and transplant network data were used to identify all patients listed for heart transplant between 2006 and 2014 within a one year period before and after their 18 th birthday. The primary study endpoint was the waiting time to receive a donor heart. Secondary endpoints included the probability of transplant and waitlist mortality. Regression discontinuity analysis was used to analyze the effect of age on either side of the sharp cut-off value of age 18 years (6,574 days of life), when allocation of donor hearts transitions from the pediatric to adult allocation system. Results A total of 360 patients met the study inclusion criteria, including 207 (57.5%) listed during the 12-month period prior to the 18 th birthday under the pediatric allocation system, and 153 (42.5%) listed during the 12 months after the 18 th birthday under the adult allocation system. The pediatric cohort was more likely to be listed status 1A. Otherwise, the two groups shared similar baseline characteristics. Overall, patients listed after the 18 th birthday waited 8.5 months longer to receive a transplant than adolescents listed before the 18 th birthday (P=0.01), had a 47% lower probability of receiving a transplant (P=0.001) but no difference in waitlist mortality (P=0.37). Conclusion Patients listed for heart transplant shortly after their 18 th birthday have significantly longer wait-times compared to patients listed shortly before their 18 th birthday, a lower probability of transplant but no significant difference in waitlist mortality. For medically fragile adolescents at high risk of death, birth date may be one factor to be aware of in timing of heart transplant listing.

Purpose Several centers have considered the potential benefits of atrial inflow cannulation to provide mechanical circulatory support (MCS) among pediatric patients with small or restrictive ventricles particularly in those with potential for myocardial recovery. Limited pediatric data are available on contemporary outcomes of atrial cannulation. We sought to describe the characteristics, complications, and outcomes of children supported with atrial cannulation using the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) ventricular assist device (VAD) database. Methods Data were collected from the ACTION VAD database for all patients who underwent VAD implantation via atrial cannulation from 8/16/2012 to 9/8/2020. Patient characteristics, VAD types, complications, and outcomes were analyzed. Results Forty-one patients met the study criteria with 52 implants. Median age was 3.6 years (range 0.02 - 25.32), weight 13.6 kg (2.7-106), and BSA 0.6 (0.15 - 2.2). Diagnoses included dilated cardiomyopathy (42%), congenital heart disease (37%), transplant graft dysfunction (7%), restrictive and hypertrophic cardiomyopathies (5% each). VAD indications were bridge to decision (42%), bridge to transplant (42%), and bridge to recovery (17%). Eighty-four percent of patients were INTERMACS 1 or 2, 44% were on ECMO, and 63% were mechanically ventilated. Devices utilized included Berlin Heart Excor (40%), paracorporeal continuous flow devices (38%), Heartware (19%) and one Heartmate 3. Median support duration was 43.5 days (range 0-307). Overall mortality was 22%. Twenty patients (49%) were successfully transplanted, 7 (17%) recovered, and 5 (12%) are still alive on the device. Adverse events included major bleeding (24%), stroke (12%), sepsis (10%), right heart failure requiring RVAD (5%), and one instance of pump thrombosis. Conclusion Atrial cannulation in pediatric MCS can be performed across a variety of anatomic substrates and age ranges with outcomes and complication rates that are comparable to left ventricular cannulation strategies. This method may be advantageous in certain circumstances including patients with small or restrictive ventricles, potential for myocardial recovery, or multiple prior sternotomies to minimize surgical dissection. Further studies are needed to delineate the ideal patient population and benefits of varying atrial cannulation techniques.

Purpose We sought to characterize strokes in children supported with ventricular assist devices (VADs) in the Advanced Cardiac Therapies Improving Outcomes Network (ACTION). Methods All patients entered in the ACTION registry (4/1/2018-6/8/2020) were included. Stroke was defined by pre-specified criteria and categorized as hemorrhagic or ischemic in etiology. All strokes were adjudicated by the data coordinating center and the adjudication committee which is comprised of members from multiple sites. Results Of 270 patients in the ACTION registry, 26 patients (9.6%) experienced 28 strokes (25 ischemic and 3 hemorrhagic). Of these 26 patients, 12 (46%) were ≤1yo, 12 (46%) had congenital heart disease, 8 (26%) had preimplant ECMO use, 23 (88%) were on paracorporeal VADs and 3 (15%) had intracorporeal VADs. The median time to stroke was 17.5 days. There were 11 (39%) and 17 (60%) strokes that occurred within 7 and 30 days, respectively, and all strokes occurring ≤30 days post-implant were ischemic. On paracorporeal VADs, there were 23 ischemic (EXCOR=9, Centrimag=6, Pedimag=6, Rotaflow=2) and 2 hemorrhagic (EXCOR=1, Centrimag=1) strokes. With intracorporeal support there were 2 ischemic (HVAD=1, HM3=1) and 1 hemorrhagic (HVAD=1) strokes. The overall stroke rate was 145.4 per 10,000 patient-days. Mortality rates were significantly higher for patients who did (10/26; 38.5%) vs. those who did not (32/144; 13.1%) experience a stroke (p Conclusion Although the stroke rate in children on VADs has decreased over time, strokes still occur in 1/10 patients and are associated with significantly increased mortality. The large majority of strokes are ischemic in nature, associated with paracorporeal VADs, and occur within the first 30 days after implant. About half of the children experiencing strokes on a VAD are ≤1yo and/or have congenital heart disease. Based on these data, ACTION is focusing ongoing quality improvement projects on decreasing strokes for children on paracorporeal devices with particular attention to the early post-implant period.

Purpose Ventricular assist device (VAD) use in patients with congenital heart disease continues to increase. However, the outcomes of supporting a Fontan circulation with a VAD are largely unknown. Methods This is a retrospective review of the Pedimacs and Intermacs databases. 55 patients underwent systemic VAD implant to support the Fontan circulation between September 2012 and December 2019. Results The mean age of patients at implantation was 12.2 ± 8.0 yrs, and mean weight, 38.1 ± 26.8 kgs. Patient demographics are presented in Table 1. 28 more VADs were implanted in 2018-2019 than in 2012-2017 (28 vs 27, P = 0.007, Figure 1). The later era had higher GFR (101.1 ± 48.5 vs 71.2 ± 34.9 P = 0.02) and were more likely being followed for heart failure prior to VAD (96% vs 74% P = 0.02), but there was no difference in INTERMACS profile (P = 0.69). Kaplan-Meier survival on device was 76% at 6 months with no difference by era. Competing outcomes demonstrated a positive outcome of 81% (alive on VAD, transplanted or recovered) at 6 months, with the majority of mortality occurring in month one. Mean length of support was 5.5 ± 8 mos. 5 patients were supported for >1 yr with no added mortality; the longest support time was 4 yrs 7mos. Adverse event rates included pump thrombosis incidence of 4% (3.3/100 pt-mo), GI bleeding of 7% (2.6/100 pt-mo), and non-GI bleeding of 9% (2.3/100 pt-mo). Freedom from stroke and infection were both 95% at 6 months. Conclusion This is the largest reported analysis of systemic VAD support of Fontan patients. Mechanical circulatory support of the Fontan circulation is increasing in prevalence and can be performed with promising outcomes.

Purpose Given the paucity of data on quality of life (QOL) after ventricular assist device (VAD) implant in children, we sought to describe self-reported QOL in children on VAD support and to identify potential factors associated with impaired QOL. Methods There were 82 children (6-19 y) in the Pediatric Interagency Registry for Mechanical Circulatory Support who completed the generic Pediatric Quality of Life Inventory (PedsQL) +/- a VAD-specific QOL assessment pre-VAD implant (n=18), 3 months (m) post-VAD (n=62) and/or 6 m post VAD (n=38). Their QOL was compared to the general pediatric population, children with a chronic health condition, and children with severe/complex heart disease. Significantly impaired QOL is a score >1 SD below the pediatric population mean. Results The study group was 59% male, 67% Caucasian, with a primary diagnosis of cardiomyopathy in 82%, and a median age at 1st implant of 14 y (IQR 11-17). At 3 m and 6 m post-VAD, 63% and 81% were outpatient. PedsQL scores were lower than pediatric norms for physical (p 2 weeks/ongoing post-VAD (p =.04), but was not associated with age at implant, primary diagnosis, hospital length of stay, adverse events or re-hospitalizations within 3 m post-implant, or with discharge status at 3 m post-VAD. Conclusion Physical QOL is significantly impaired in the majority of patients pre- and post-VAD. However, psychosocial QOL is not significantly impaired in nearly 3/5 patients at 3 m post-VAD. While the study was limited by the small number of patients who had more than one assessment over time, group data suggest VAD implantation may improve psychosocial QOL.

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.

Purpose Individuals born with defects resulting in a functionally univentricular heart Fontan-type palliation (FP) are prone to physiologic deterioration requiring advanced cardiac therapies. Given limitations to organ availability and long transplant waiting-list times, mechanical circulatory support (MCS) has become an important tool to permit individuals with a single ventricle circulation to either survive to transplant or may be used for destination therapy. The physiologic and clinical impact of MCS in FP patients are undefined. Methods Centers participating in the ACTION Learning Network entered data on FP patients that received a VAD at their center, between 1/26/12 and 10/1/2019, in the ACTION Outcomes registry. Additional survey data on physiologic variables will be completed by Nov 2019. Results Thirty-nine FP patients were reported to the ACTION registry. Limited clinical variables can be found in table 1. MCS types included intracorporeal continuous flow support (25 patients), paracorporeal support (10), total artificial heart (2) and biventricular assist devices (1). Among supported patients, 23 % (9) died while on support, 10 % (4) are currently alive on support, and 67% (26) were transplanted and are alive while none died post-transplant. Major adverse events included major bleeding (17 episodes), driveline infection (n=1) and other neurologic dysfunction (n=1). There was no stroke or pump thrombosis (table 1). MCS implant improved NYHA functional class (12.5% versus 75% NYHA class 1/2) and decreased severe atrioventricular valve regurgitation (47% versus 30%). Complete clinical and hemodynamic data will be available and presented at the ISHLT meeting. Conclusion This is the first multicenter report of FP outcomes post MCS. MCS results in improved functional status and decreased atrioventricular valve regurgitation in FP patients awaiting transplant without adverse post-transplant outcomes and with good on-device survival. MCS for Fontan patients as both destination and bridge to transplant is a reasonable option.

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.

The utility of hemodynamic ramp studies in single ventricle patients supported by continuous flow left ventricular assist devices (CF-LVADs) is largely unknown. We report the novel use of the hemodynamic ramp test to optimize a 26 year-old Fontan-palliated single-ventricle patient supported with a continuous-flow ventricular assist device (Heartware HVAD). Modest improvements in Fontan hemodynamics resulted in significant and persistent clinical improvement and symptomatic relief.

Due to limited and conflicting data in pediatric patients, long-term routine surveillance endomyocardial biopsy (RSB) in pediatric heart transplant (HT) remains controversial. We sought to characterize the rate of positive RSB and determine factors associated with RSB-detected rejection. Records of patients transplanted at a single institution from 1995 to 2015 with >2 year of post-HT biopsy data were reviewed for RSB-detected rejections occurring >2 year post-HT. We illustrated the trajectory of significant rejections (ISHLT Grade ≥3A/2R) among total RSB performed over time and used multivariable logistic regression to model the association between time and risk of rejection. We estimated Kaplan-Meier freedom from rejection rates by patient characteristics and used the log-rank test to assess differences in rejection probabilities. We identified the best-fitting Cox proportional hazards regression model. In 140 patients, 86% did not have any episodes of significant RSB-detected rejection >2 year post-HT. The overall empirical rate of RSB-detected rejection >2 year post-HT was 2.9/100 patient-years. The percentage of rejection among 815 RSB was 2.6% and remained stable over time. Years since transplant remained unassociated with rejection risk after adjusting for patient characteristics (OR = 0.98; 95% CI 0.78-1.23; P = 0.86). Older age at HT was the only factor that remained significantly associated with risk of RSB-detected rejection under multivariable Cox analysis (P = 0.008). Most pediatric patients did not have RSB-detected rejection beyond 2 years post-HT, and the majority of those who did were older at time of HT. Indiscriminate long-term RSB in pediatric heart transplant should be reconsidered given the low rate of detected rejection.

Despite significant advances in the medical field, pediatric heart failure continues to have a significant health burden. ACTION (Advanced Cardiac Therapies Improving Outcomes Network) was formed to bring the benefits of a learning health system to the problems of pediatric heart failure and VAD care. The ACTION learning collaborative network has expanded its focus to incorporate quality improvement initiatives aimed at improving outcomes in pediatric heart failure. The multiple initiatives include a collaboration with the Pediatric Health Information System (PHIS) database, timely referral for heart failure management in Fontan patients, formation of a waitlist outcomes committee, and a heart failure management committee with numerous parent and provider education initiatives.

In neonates, it is challenging to diagnose aortic coarctation in the setting of a patent ductus arteriosus (PDA). Frequently, serial echocardiograms are performed, and diagnosis is delayed until the PDA closes. The purpose of this study was to identify echocardiographic predictors of neonatal coarctation in the presence of a PDA in cases in which diagnosis is uncertain.We retrospectively identified neonates diagnosed with possible but not definitive coarctation in the presence of a PDA by echocardiography (January 2004 through August 2013). The carotid-subclavian artery index (CSAi) was defined as the distal transverse arch diameter divided by the distance between the left common carotid and left subclavian arteries. Medical records were reviewed to identify patients who underwent coarctation repair within 1 year. A separate validation group was identified with the same methodology (September 2013 through April 2015).Thirty-three patients were identified (median age 1, range 0-8 days). Twelve patients (36%) underwent coarctation repair. The coarctation group had smaller aortic and mitral valves, distal transverse arch, and isthmus z scores, larger right innominate artery, and longer transverse arch compared with the remaining group (p0.05). The CSAi was lower in the coarctation group (p = 0.014), and a cutoff of less than 0.85 yielded a sensitivity of 0.83 and specificity of 0.86 for coarctation (area under the receiver operating characteristic curve, 0.91). In the validation group (n = 12; median follow-up, 316 days), none of the 8 patients with a CSAi of greater than 0.85 have required surgery. The intraclass correlation coefficient for CSAi was 0.79 (95% confidence interval, 0.18 to 0.95).The CSAi, a simple and reproducible measure, can identify neonates at risk for aortic coarctation even in the presence of a PDA, prevent multiple echocardiographic evaluations, and hence guide appropriate resource utilization.

Purpose Ventricular assist devices (VADs) increase waitlist survival, yet the risk of stroke is not trivial. There is a knowledge gap regarding the impact of these strokes on heart transplant outcomes. We sought to analyze how strokes during VAD support impact post-transplant (post-Tx) outcomes in children. Methods 520 pediatric ( Results Overall, there were 146 (28%) VAD patients with a pre-Tx stroke; 86 (59% of stroke patients) of them were transplanted at a median of 57 [17-102] days from stroke, significantly lower than the no stroke cohort (90%; p Conclusion Patients with stroke on VAD once transplanted, enjoy similar survival compared to no stroke patients. Allowing children who suffer stroke while on VAD support to have more time to recover appears to improve post-Tx outcomes.

Purpose Ventricular assist device (VAD) interrogations allow clinicians to establish and maintain optimal VAD settings through the support course. There is currently no data regarding standards for establishing and modifying pediatric VAD settings, despite a heterogeneous population with variable diagnoses, anatomy and physiology. Methods The Advanced Cardiac Therapies Improving Outcomes Network (ACTION), a 37-center North American learning network, was surveyed in July 2019 regarding current practices for establishing, monitoring and modifying pediatric VAD settings. Results A total of 25 (68%) centers responded to the survey. Criteria for establishing intraoperative device settings varied between solely surgeon determined (36%) versus global imaging, surgeon, and VAD team driven (60%). Post-operative inpatient and outpatient interrogations were performed predominantly (80%) by a VAD physician or advanced practice provider (APP), but frequency ranged from daily (64%) to weekly (24%) to even monthly (4%). There was consensus (100%) on including device trends and alarm assessment for VAD interrogation, but many centers (88%) include log file interpretation in each interrogation as well. Factors used for optimization of post-operative settings were mixed. Several centers (28%) rely only on device interrogation and clinical course while many (24%) utilize echocardiogram only. The majority (44%) of sites use a combination of echocardiogram and device interrogation to determine optimal support. Outpatient practices were similar amongst sites with the exception of device wean trials which had significant variation with regards to use of echocardiogram (16%), hemodynamic catheterization (8%) or a combination of both studies (76%). Billing practices for VAD interrogation were inconsistent amongst centers, particularly regarding billing frequency. Conclusion VAD interrogation and optimization practices were highly variable amongst ACTION centers, most notably during inpatient hospitalizations. Given the majority of pediatric VAD patients remain hospitalized while on their device, a standardized approach to this process may minimize unnecessary variation. Furthermore, defining “standard” management approaches and device settings in varied pediatric populations may help define the optimal clinical approach to support.

Purpose Ventricular assist devices (VADs) have improved wait list survival in children with end-stage heart failure. However, questions remain about the optimal timing of VAD implantation. In this cost-effectiveness analysis, we compared immediate intracorporeal continuous VAD implantation to a watchful waiting approach using continuous inotropic therapy. Methods Using a Markov model, we simulated a cohort of pediatric patients with dilated cardiomyopathy. We used a monthly cycle and 20-year time horizon. We estimated cost and benefits (in terms of quality-adjusted life years, QALYs) from a healthcare perspective discounted at 3% per year. Transition probabilities, costs, and utility weights were taken from published and grey literature, which we supplemented with expert opinion where there were gaps. We calculated the incremental cost-effectiveness ratio (ICER) and performed one-way and probabilistic sensitivity analyses (PSA) using 10,000 Monte Carlo simulations to understand the impact of parameter uncertainty in the main findings. Cost variables were assigned gamma distributions, while transition probabilities and utilities were assigned beta distributions. Results In a base case analysis, the incremental costs and benefits of immediate intracorporeal continuous VAD implantation compared to medical management was $221,269 (2017 US$) and 0.21 QALYs, respectively, resulting in an ICER of $1.04 million per QALY. One-way sensitivity analysis found that the discount rate, the probability of heart transplant among patients on VAD and medical management, and the cost of VAD implantations exerted the most influence on the ICER, though varying any of the parameters to their lowest and highest values did not bring the ICER close a $100,000 per QALY willingness-to-pay (WTP) threshold. The PSA showed a 3% chance of immediate VAD implantation to be cost-effective at a WTP of $500,000 per QALY and a 36% chance at $1 million per QALY. Conclusion Though VADs provide a net health benefit as a bridge to transplant, immediate implantation in children with inotrope-dependent heart failure is not likely to be a cost-effective strategy compared to medical management based on commonly used ICER thresholds. Future analyses should explore the cost-effectiveness of VAD implantation in children with other disease states, diagnoses and device types.

BACKGROUND The reported ventricular assist device (VAD) experience in the pediatric congenital heart disease (CHD) population is limited. We sought to describe contemporary use and outcomes of VADs in children with CHD and compare these outcomes to those of non-CHD children. METHODS Patients enrolled in the Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) between September 19, 2012 through June 30, 2017 were included. CHD was classified as biventricular vs single ventricle (Stages 1, 2, or 3). Outcomes were compared between groups and multivariable analysis was used to identify factors associated with mortality on the device. RESULTS Among the 471 patients enrolled, 108 (24%) had CHD (45 biventricular and 63 single ventricle). CHD patients were younger (5.7 ± 5.7 years vs 9.8 ± 6.5 years; p < 0.0001) and smaller (0.8 ± 0.5 m2 vs 1.2 ± 0.7 m2; p < 0.0001) compared with non-CHD patients. CHD patients were more likely to receive a paracorporeal continuous-flow VAD (36.1% vs 12.9%; p < 0.0001) and less likely to receive an implantable continuous-flow VAD (27.8% vs 55.0%; p < 0.0001) compared with non-CHD patients. After 6 months on a VAD, CHD patients had higher mortality (36.4% vs 12.1%) and a lower transplantation rate (29.1% vs 59.9%) than non-CHD patients (p < 0.0001). In the multivariable analysis, CHD was the factor most strongly associated with mortality on VAD (hazard ratio [HR] = 2.9; p < 0.0001), whereas the factors implantable continuous-flow device and high-volume center were protective (HR = 0.3, p < 0.0001, and HR = 0.6, respectively; p = 0.02). CONCLUSIONS VAD use in children with CHD is associated with increased mortality and decreased transplant rates compared to children without CHD. For the subgroup of children with CHD who received implantable continuous-flow VADs, survival rates were higher and comparable to those of children without CHD. Increased experience correlated with better survival in pediatric VADs.

Purpose Elevated blood pressure (BP) is a risk factor for stroke in adult ventricular assist device (VAD) patients. There are currently no data or BP management guidelines for pediatric VAD patients. ACTION ( Advanced Cardiac Therapies Improving Outcomes Network) is the first pediatric VAD quality improvement network. The initial goal of ACTION is stroke reduction. This report describes the methods used to obtain BP and effectiveness of BP control among ACTION members. We also describe the development of a BP clinical decision support (CDS) tool to standardize BP reporting and goals. Methods The BP protocols among ACTION members were assessed. Process measures that may impact stroke rates were identified via anonymous surveys beginning June 2018. Statistical process control charts were used to assess baseline metrics. Results A total of 28 patients from 11 centers were identified with BP data. Including 12 Heartware, 10 Berlin, 2 Heartmate3, and 4 paracorporeal continuous flow (CF) devices. Height/age BP %iles were used to guide BP goal in 19 (68%) patients. Among CFVAD patients, BP measurement method (outside the perioperative period) and BP goal value varied, especially among the 11 patients (55%) with a palpable pulse (Figure 1). Patients had well controlled BP on 69% of support days (Figure 2). Conclusion The BP measurement methods and goals varied across ACTION, especially among patients on a CF-VAD with a pulse. A CDS tool has been developed to standardize BP measurement and management among ACTION members and will guide subsequent collaborative studies to assess the role of BP in stroke among pediatric VAD patients.

Purpose Pediatric Ventricular Assist Device (VAD) therapy is associated with high rates of stroke and bleeding. There have been limited efforts to date, to define and implement optimal anticoagulation (AC) strategies in this population. ACTION (Advanced Cardiac Therapies Improving Outcomes Network) is the first pediatric VAD quality improvement network, with an initial goal of stroke reduction. This report describes baseline AC data from ACTION centers, including agents used and aggregate levels of performance, to inform development of specific interventions. Methods The AC practices of ACTION centers were assessed. Data collected included VAD type, weekly data characterizing AC agents used; time to achieve desired levels; and percentage of levels within the desired range. Results Data were collected from 11 centers, with a total of 30 patients. AC data were collected from 6/1-9/1/18, while strokes were collected retrospectively for the entire VAD support interval. Devices included: HVAD (12); EXCOR (10); Heartmate 3 (3); and 5 paracorporeal continuous flow devices. There were 5 strokes (16.7%). Intravenous AC included unfractionated heparin (36%), and bivalirudin (64%). There was wide variability between patients in the time to achieve first therapeutic AC level (Figure 1), with 15 patients at goal within 20 hours, while others required up to 170 hours. Heparin levels varied widely, with the percent of labs within range falling below 50%, in 50% of the weeks reported. For bivalirudin, levels were less variable, with percent of labs within range falling below 50% in only 14% of weeks reported. Conclusion At baseline, stroke rates remain high in the ACTION centers. Intravenous AC includes both heparin and bivalirudin, with high variability in time until therapeutic levels are achieved, and with high variability in subsequent levels, particularly for heparin. These data will inform interventions designed to reduce variability in AC performance as a basis for stroke reduction.

A previously healthy male infant born at 39 weeks’ gestation to a 33-year-old, gravida 1 para 1 mother presents to an outside hospital with respiratory distress at age 10 days. The infant was delivered via cesarean delivery for failure to progress, with Apgar scores of 8 and 9 at 1 minute and 5 minutes, respectively. His nursery stay was uneventful, and he was discharged home on day 2 after birth. During the next several days, he developed feeding difficulty and increased work of breathing. At the outside hospital emergency department, rectal temperature is 98.8°F (37.1°C), heart rate is 170 beats per minute, blood pressure is 105/74 mm Hg, respiratory rate is 78 breaths per minute, and oxygen saturation is 84% on room air. Reported physical examination findings are otherwise unremarkable. Continuous positive airway pressure with pressure support breaths is initiated. Electrocardiography reveals sinus tachycardia. Initial laboratory evaluation reveals the following: pH 6.8; bicarbonate, 7 mEq/L (7 mmol/L); base deficit, −27 mEq/L (−27 mmol/L); lactate, 153 mg/dL (17 mmol/L); anion gap, 29 mEq/L (29 mmol/L); white blood cell count, 27,000/μL (27.0 × 109/L), with normal differential; hematocrit, 57% (0.57); C-reactive protein, 1.0 mg/L (9.5 nmol/L); and glucose, 39 mg/dL (2.2 mmol/L). The patient is intubated. Chest radiography is performed (Fig 1). Dextrose, normal saline, bicarbonate, and broad-spectrum antibiotics are given for presumed sepsis, and the patient transfers to our neonatal intensive care unit for further evaluation and management. Figure 1. Chest radiograph of the …

The coronary sinus can become obstructed with any instrumentation at or near the ostium such as in atrioventricular canal defect repairs. This complication may lead to a wide range of consequences including dyspnea, angina, myocardial infarction, and sudden death. The following report illustrates the importance of careful perioperative echocardiographic evaluation of the coronary sinus in procedures that may affect the sinus and its ostium.