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For smaller pediatric patients on ventricular assist devices, the Berlin Heart EXCOR remains the main form of durable support. It requires a connection to the external IKUS, which has limited portability and battery life. The new EXCOR Active mobile driving unit has a battery life of up to 13 hours. We describe the first North American experience with the EXCOR Active in pediatric patients with a Berlin Heart device. A retrospective chart review was undertaken. Between October 2022 and March 2024, 7 patients were on a Berlin Heart and supported with the EXCOR Active. All patients were initially supported with the IKUS with a median time to transition to the EXCOR Active of 12.0 days (interquartile range [IQR] 9.5, 18.5) and a median time of support with the EXCOR Active of 65.0 days (IQR, 32.0, 81.0). The EXCOR Active posed no significant safety issues, and minimal operating issues were noted. Following the transition from IKUS to the EXCOR Active, there was increased patient and caregiver mobility throughout the hospital. Use of the EXCOR Active has the potential to improve the quality of life in pediatric patients waiting for heart transplantation. [ABSTRACT FROM AUTHOR]

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, 2022, 1463 devices in 1219 patients aged <19 years were reported to the registry from 40 North American hospitals. Cardiomyopathy remains the most common underlying etiology (59%), followed by congenital heart disease (26%) and myocarditis (8%). Implantable continuous devices were most common (39%) type, followed by paracorporeal pulsatile (28%) and paracorporeal continuous (27%) devices. At 6 months after VAD implantation, a favorable outcome (transplant, recovery, or alive on device) was achieved in 85% of patients, which was greatest among those on implantable continuous VADs (92%) and least for paracorporeal continuous VADs (68%), although the patient population supported on these devices is different. This Seventh Pedimacs Report demonstrates the continued importance of VADs in the treatment of children. With the complexity of cardiac physiologies and sizes of patients, multiple types of devices are used, including paracorporeal continuous, paracorporeal pulsatile, and implantable continuous devices. The preoperative risk factors and differences in patient populations may account for some of the differences in survival observed among these devices. This report, along with other collaborative work, continues to advance the care of this challenging and vulnerable population. [ABSTRACT FROM AUTHOR]

The removal of the HeartWare ventricular assist device (HVAD) due to pump malfunctions and inferior outcomes compared to HeartMate 3 (HM3) in adults has created a care gap for younger patients. It is unclear if the reported HVAD survival differs by age and if the initial experience with HM3 can bridge the gap. Using the Society of Thoracic Surgeons (STS) Intermacs and Pedimacs registries, durable ventricular assist device (VAD) implants between September 2012 and December 2021 were identified. Young adults (YA) were defined as <40 years old in Intermacs. Patients were excluded if they had an isolated right VAD (RVAD) or were implanted as destination therapy (DT). Survival analysis by Kaplan-Meier (KM) and competing outcomes curves was performed, and 1-year survival is reported. The Intermacs cohort consisted of YA (n = 1226; HVAD 818; HM3 408) with a median age of YA of 32.07 (26.66-36.27) years and weight (wt) of 83.2 (68-104.2) kg. Most had cardiomyopathy (CM) (92.2%). The Pedimacs cohort was 668 patients (median age 9.47 [1.82-14.23] years, wt 27.2 [10-57.05] kg), and most also had CM (70.5%). Device breakdown included HVAD (n = 326), Berlin EXCOR (n = 277), and HM3 (n = 65). HVAD survival differed by age in adults, with YA fairing better than adults > 40 years old (88.8% vs 79.4% at 1 year, p < 0.0001). YA survival was also better compared to Pedimacs patient (88.9% vs 83.7%, p = 0.0002), but when competing events were analyzed, mortality was similar to YA (9.2% vs 9.6%, p = 0.1) with a higher proportion of patient undergoing transplant at 1 year in Pedimacs (74% vs 31.3%, p < 0.0001). Survival by device differed between HVAD and HM3 in YA (88.8% vs 94.4%, p = 0.0025). This difference in device survival was not seen in all children (83.7% vs 87.3%, p = 0.21), including those ≥25 kg. Adverse event profiles also differed across the groups with adults seeing less adverse events with the HM3, but the same was not found (including stroke) in the pediatric cohort. Survival outcomes for patients between 10 and 25 kg were similar with the HVAD compared to the Berlin Heart EXCOR (p = 0.4290), with similarities in stroke risk. The removal of the HVAD device may result in a care gap in younger patient whose survival outcomes do not mirror that of older adults. The HM3 can fill a portion of this gap with good survival, but there remains a subset of pediatric patients that, based on initial HM3 use, will no longer have access to intracorporeal support and therefore, despite reasonable outcomes with the Berlin Heart EXCOR, will not be able to be discharged home. Lastly, it is essential that future changes to the availability of devices take into account the various patient populations that utilize the device to avoid unintended consequences of access inequality. [ABSTRACT FROM AUTHOR]

Ventricular assist devices are important in the treatment of pediatric heart failure. Although paracorporeal pulsatile (PP) devices have historically been used, there has been increased use of paracorporeal continuous (PC) devices. We sought to compare the outcomes of children supported with a PP or PC, or combination of devices. A retrospective review (2005 to 2019) was made of patients less than 19 years of age from a single center who received a PC, PP, or combination of devices. Patient characteristics were compared between device strategies, and Kaplan-Meier survival analysis was performed. Sixty-six patients were included: 62% male; 62% non-congenital heart disease; median age 0.9 years (interquartile range, 0.2 to 4.9); and median weight 8.5 kg (interquartile range, 4.3 to 17.7 kg). The PC devices were used in 45% of patients, PP in 35%, and a combination in 20%. Patients on PC devices had a lower median weight (P =.02) and a higher proportion of congenital heart disease (P =.02), and more patients required pre-ventricular assist device dialysis (P =.01). There was no difference in pre-ventricular assist device extracorporeal membrane oxygenation use (P =.15). There was a difference in survival among the three device strategies (P =.02). Differences in survival were evident, with patients on PC support having worse outcomes. Transition from PC to a PP devices was associated with a survival advantage. These findings may be driven by differences in patient characteristics across device strategies. Further studies are required to confirm these findings and to better understand the interaction between patient characteristics and device options. [ABSTRACT FROM AUTHOR]

We sought to evaluate the association between timing of cardiac allograft vasculopathy (CAV) occurrence post-heart transplant (HT) with graft survival and progression of CAV severity in pediatric HT recipients. Data from the Pediatric Heart Transplant Society for pediatric (<18 years old) HT recipients between 1993-2019 with available angiographic data were obtained (N = 5,075). The timing of CAV diagnosis (<3; 3-<5; 5-<10; and ≥10 years post-HT) and severity of disease at each assessment (CAV 1-3) was determined. Associations between CAV timing, graft survival, and CAV progression were evaluated using Kaplan-Meier survival curves, multivariable COX proportional hazard regression analyses, and competing risk analyses. Over a median follow-up period of 4.1 (IQR 1.3-8.3) years, CAV was identified in 17% (885/5,075), 28% (252/885) of which were early-onset CAV. Compared with late onset CAV ≥10 years post-HT, patients with early CAV were older at the time of transplant (8.3 ± 6.2 vs. 3.8 ± 4.8 years, p <.0001). While the five-year graft-survival in the ≥10-year group (79.2%, p = 0.03) was significantly higher than the <3, 3-<5, and 5-<10 years post-HT groups (65.0%-67%) (p = 0.03), overall post-CAV graft survival was not significantly different across the CAV time-points. CAV disease progression did not vary with CAV timing post-HT, with an overall five-year freedom from CAV ≥2 of 75.4% (73.1%-77.6%). Later onset CAV (≥10-years post-HT) was associated with improved five-year graft survival compared with CAV onset at earlier time-points, but similar and poor long-term outcomes. CAV timing post-HT was not associated with progression of CAV disease severity. [ABSTRACT FROM AUTHOR]

Reports focused on adult heart transplant (HTx) recipients with COVID-19 suggest an increased risk of severe disease, however; it is unclear if this holds true for pediatric HTx patients, given the typically milder course of illness in children in general with COVID-19. We sought to rapidly implement a system for multi-center data collection on pediatric HTx candidates and recipients, with the aim of describing the patient population and infection related outcomes. The Pediatric Heart Transplant Society (PHTS) is a multi-center collaboration that seeks to improve the outcomes of children who are listed and undergo HTx. The society consists of pediatric HTx centers in North America (n = 53), UK (n = 2), and Brazil (n = 1). In response to the pandemic, PHTS developed a web-based platform to collect COVID-19 specific data on pediatric HTx candidates and recipients. Non-PHTS centers were also invited to submit data. Data fields included pre-and post-HTx patient characteristics, presumed versus documented infection, need for hospitalization (including ICU and ventilator use), treatments administered, and 30-day outcome (resolution, death, sequelae, and or unresolved) Data collection was initiated on 4/30/20. As of 03/15/21 there were 225 patients [19 pre-HTx and 206 post-HTx, median age 14 years (IQR 7, 18)] reported from 41 centers. Hospitalization occurred in 42% (n = 8) of the pre-HTx and 21% (n=43) of the post-HTx patients. Among the patients listed for HTx, 21% (n = 4) required ICU and 10.5% (n = 2) were mechanically ventilated. Among post-HTx patients, 7% (n = 14) required ICU and 1% (n = 3) were mechanically ventilated. At 30 days, the majority of patients had resolution of symptoms (94.7% pre-HTx, 95.6% post-HTx). One death was reported in a post-HTx patient prior to 30 days from onset of COVID-19 illness. These data demonstrate the ability to rapidly adapt the PHTS data collection infrastructure in response to a novel infection and represent the first known multi-center report of characteristics and early outcomes for patients listed and following pediatric HTx with COVID-19. Hospitalization appears to be more common for both candidates and recipients due to COVID-19 than for the general pediatric population though stays were short and mortality minimal. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. 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). 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. 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. [ABSTRACT FROM AUTHOR]

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. Between September 19, 2012, and December 31, 2019, 44 hospitals implanted 1031 devices in 856 patients under 19 years of age. 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. 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. [ABSTRACT FROM AUTHOR]

The primary objectives of this study were to describe the characteristics and survival outcomes for children supported with the HeartWare HVAD® system from the global community. This was a retrospective survey of patients <18 years of age with an HVAD® system. Questionnaires were sent to sites worldwide in April 2015 and collected between May 2015 and 2016. Information on 205 patients was collected. The median age at implantation was 13.1 years (interquartile range [IQR] 9.8-15.8 years) and the weight was 42 kg (IQR 28-60 kg). Over half of the implants occurred in males (61%), with the most common diagnosis being cardiomyopathy (n = 168, 82%). The majority of HVAD® systems implanted were left ventricular assist device (n = 189, 92.2%). Temporary right ventricular support was utilized in 24 patients (12%) with a median duration of 12 days (6-32 days). Fifty-five percent (n = 111) of the patients were discharged home after implantation after a median (IQR) duration of hospital stay of 40 days (28-71 days). By 12 months, the proportion of patients who underwent heart transplant was 65.4%, 10.7% had died, 3.2% were explanted for recovery, and 20.8% remained on the device. Death on the device on multivariable analysis was associated with the need for temporary RV support (hazard ratio [HR] 10.65 (95% CI 12.53-44.81), P = 0.001) and pump exchange (HR 7.9 (95% CI 1.8-34.2], P = 0.006). The use of the HeartWare HVAD system in the pediatric population is associated with mortality with the majority of patients supported to heart transplant by 1 year post implant. These positive results are independent of geographic location. The need for a temporary right heart support and pump exchange is associated with a higher risk of poor outcomes, and further work is required to predict these patients to allow for optimization. Although survival results are promising, further studies are needed to delineate the associated morbidities with this technology in the pediatric population. [ABSTRACT FROM AUTHOR]

Background Short-term continuous-flow ventricular assist devices (STCF-VADs) are increasingly being used in the pediatric population. However, little is known about the outcomes in patients supported with these devices. Methods All pediatric patients supported with a STCF-VAD, including the Thoratec PediMag or CentriMag, or the Maquet RotaFlow, between January 2005 and May 2014, were included in this retrospective single-center study. Results Twenty-seven patients (15 girls [56%]) underwent 33 STCF-VAD runs in 28 separate hospital admissions. The STCF-VAD was implanted 1 time in 23 patients (85%), 2 times in 2 patients (7%), and 3 times in 2 patients (7%). Implantation occurred most commonly in the context of congenital heart disease in 14 runs (42.2%), cardiomyopathy in 11 (33%), and after transplant in 6 (18%). The median age at implantation was 1.7 (interquartile range [IQR] 0.1, 4.1) years, and median weight was 8.9 kg (IQR 3.7, 18 kg). Patients were supported for a median duration of 12 days (IQR 6, 23 days) per run; the longest duration was 75 days. Before implantation, 15 runs (45%) were supported by extracorporeal membrane oxygenation (ECMO). After implantation, an oxygenator was required in 20 runs (61%) and continuous renal replacement therapy in 21 (64%). Overall, 7 runs (21%) resulted in weaning for recovery, 14 (42%) converted to a long-term VAD, 4 (12%) resulted in direct transplantation, 3 (9%) were converted to ECMO, and 5 (15%) runs resulted in death on the device or within 1 month after decannulation. The most common complication was bleeding requiring reoperation in 24% of runs. In addition, 18% of runs were associated with neurologic events and 15% with a culture-positive infection. Hospital discharge occurred in 19 of 28 STCF-VAD admissions (67%). In follow-up, with a median duration of 9.2 months (IQR 2.3, 38.3 months), 17 patients (63%) survived. Conclusions STCF-VADs can successfully bridge most pediatric patients to recovery, long-term device, or transplant, with an acceptable complication profile. Although these devices are designed for short-term support, longer support is possible and may serve as an alternative approach to patients not suitable for the current long-term devices. [ABSTRACT FROM AUTHOR]

Failing single-ventricle patients have now come into focus as the next cohort where improvement in outcomes for mechanical circulatory support can be realized. There is a paucity of published patient reports or management protocols in this patient population. Increased interest exists in finding answers of how to bridge these patients to transplant. We review the current literature and describe our approach to the patient with univentricular heart needing mechanical circulatory support. [ABSTRACT FROM AUTHOR]

Objective: Despite improved long-term survival after pediatric heart transplantation, perioperative mortality has remained high. We sought to understand the factors associated with perioperative graft loss after pediatric heart transplantation. Methods: The factors associated with primary heart transplant mortality and retransplantation before hospital discharge in 226 pediatric heart transplant recipients (1995-2010) at a single-center institution were analyzed using multivariable logistic regression models adjusted for age at surgery and year of surgery. Results: A total of 26 patients died (n = 21) or underwent retransplantion (n = 5) before hospital discharge secondary to primary graft failure (n = 10), multisystem organ failure (n = 5), infection (n = 4), rejection (n = 2), and perioperative complications (n = 5). United Network for Organ Sharing status 1 (vs status 2) at transplantation was associated with an increased odds of death from noncardiac causes (odd ratio [OR], 4.7; 95% confidence level [CI], 1.2-22.3; P = .002). The factors associated with increased odds of perioperative mortality or retransplant were pre- and post-transplant extracorporeal membrane oxygenation (OR, 5.3; 95% CI, 1.5-18.7; P = .01; and OR, 25.9; 95% CI, 7.0-95.9; P < .001), longer ischemic times (OR, 1.4 per 30 minutes; 95% CI, 1.0-2.0; P = .04), reoperation after transplantation (OR, 3.5; 95% CI, 1.2-10.4; P = .02), and transplantation before 2002 (OR, 4.5; 95% CI, 1.4-14.9; P = .01), respectively. Conclusions: The use of extracorporeal membrane oxygenation (both before and after transplantation), a longer ischemic time, and reoperation were key factors associated with perioperative graft loss, with noncardiac mortality closely related to United Network for Organ Sharing status at heart transplantation. Knowledge of the perioperative risk factors and how they affect graft survival will help guide difficult decisions around eligibility, timing of primary listing, and appropriateness for retransplantation, and potentially affect long-term survival. [Copyright &y& Elsevier]

Background: Retransplantation for graft failure in the pediatric population is rare with reports of decreased survival compared with primary transplantation. Little is known about the risk factors for retransplantation or death after retransplantation; in addition, there is a paucity of data in the literature on transplant-related morbidities in this patient population. The purpose of this analysis of the registry of the International Society of Heart and Lung Transplant (ISHLT) was to describe outcomes after retransplantation as compared with primary transplantation, including identifying risk factors leading to retransplantation and both transplant-related morbidities and mortality after retransplantation. Methods: Data from 1998 to 2010 were used to identify all patients whose primary transplantation was performed at <18 years of age. Of the 9,966 transplants reviewed, 9,248 primary transplants and 602 retransplants were analyzed. Results: The median age for retransplantation was 14 (range 1 to 26) years, with the most common indication being allograft vasculopathy (58.5%). After retransplantation, early time-related risk of mortality was similar to that after primary transplantation (HR 1.07 [0.92 to 1.25], p = 0.40), but both late-phase time-related risk of mortality (HR 1.67 [1.40 to 1.99], p < 0.001) and requirement of an additional graft (HR 1.69 [1.18 to 2.43], p = 0.004) were higher. Long-term morbidities were significantly more common after retransplantation than with primary transplantation. These included allograft vasculopathy (HR 2.8 [2.37 to 3.38], p < 0.001), late rejection (HR 2.0 [1.65 to 2.40], p < 0.001) and late renal dysfunction (HR 2.6 [2.18 to 3.12], p < 0.001), but not cancer (HR 1.16 [0.74 to 1.82], p = 0.52). Numerous patient-, donor- and transplant-related risk factors were identified that increased both the risk of mortality and transplant-related morbidities after retransplantation. Conclusions: Retransplantation after primary transplant in the pediatric age group, although feasible with similar early survival, is associated with decreased long-term survival and an increase in transplant-related morbidities. The results of this study highlight the ongoing challenges of managing patients after retransplant and also suggest some modifiable risk factors, for both the donor and recipient, which may improve survival and decrease the burden of morbidities. [Copyright &y& Elsevier]

Abstract: Cardiomyopathies are the primary indication for transplantation in children over one year of age. Transplantation offers excellent short and intermediate term survival. However, there continues to be a number of patients who die awaiting transplantation due to the ongoing challenges with donor organ availability. This chapter will explore general indications for transplantation in children with cardiomyopathies, the waitlist mortality in this patient population and risk factors for delisting or death while waiting. [Copyright &y& Elsevier]

Sudden death after pediatric cardiac transplant is a rare and devastating event. We report the case of a young girl who died suddenly with an unexplained persistent peripheral blood eosinophilia after cardiac transplantation. [ABSTRACT FROM AUTHOR]

Background: The impact of the 2007 American Heart Association endocarditis prophylaxis guidelines on clinician practice has not been well established. Our objective was to evaluate how the American Heart Association endocarditis guidelines changed the practice of cardiologists who manage congenital heart disease and to ascertain the degree of practice variation among cardiologists. Methods: A cross-sectional Web-based survey was e-mailed to Canadian (n = 134), Australian (n = 33), New Zealand (n = 9), and a random sample of American (n = 250) pediatric and adult congenital heart disease cardiologists in 2008. Nonrespondents received the survey 4 times by e-mail and once by regular post. Results: The response rate was 55%. The lesions for which cardiologists were most evenly divided between recommending versus not recommending prophylaxis were “rheumatic mitral stenosis of moderate severity” (45% recommended prophylaxis) and “perimembranous ventricular septal defect (VSD) status post surgical patch closure with no residual shunt 3 months post-operatively” (54% recommended prophylaxis). The lesions for which the greatest proportion of cardiologists discontinued prophylaxis were “small muscular VSD, no previous endocarditis” (80% discontinued prophylaxis) and “small audible patent ductus arteriosus” (83% discontinued prophylaxis). Only 69% recommended prophylaxis for “VSD s/p surgical patch closure with small residual shunt” despite current guidelines recommending prophylaxis for this scenario. Twenty-eight percent of respondents felt that the new guidelines leave some patients at risk, and 6% would not recounsel any low-risk patients following these guidelines. Conclusions: The 2007 guidelines have resulted in a substantial change in endocarditis prophylaxis. There remains considerable heterogeneity among cardiologists regarding the prophylaxis of certain cardiac lesions. [Copyright &y& Elsevier]

Hurler syndrome is a lysosomal storage disease resulting in fatal cardiac or neurologic sequelae unless alpha-iduronidase production is reconstituted with hematopoietic stem cell transplantation. We report on a 4-year, 6-month-old boy with mixed donor chimerism and low enzyme levels but a normal neurodevelopmental trajectory. [Copyright &y& Elsevier]

There is no FDA-approved left ventricular assist device (LVAD) for smaller children permitting routine hospital discharge. Smaller children supported with LVADs typically remain hospitalized for months awaiting heart transplant—a major burden for families and a challenge for hospitals. We describe the initial outcomes of the Jarvik 2015, a miniaturized implantable continuous flow LVAD, in the NHLBI-funded Pumps for Kids, Infants, and Neonates (PumpKIN) study, for bridge-to-heart transplant. Children weighing 8 to 30 kg with severe systolic heart failure and failing optimal medical therapy were recruited at 7 centers in the United States. Patients with severe right heart failure and single-ventricle congenital heart disease were excluded. The primary feasibility endpoint was survival to 30 days without severe stroke or non-operational device failure. Of 7 children implanted, the median age was 2.2 (range 0.7, 7.1) years, median weight 10 (8.2 to 20.7) kilograms; 86% had dilated cardiomyopathy; 29% were INTERMACS profile 1. The median duration of Jarvik 2015 support was 149 (range 5 to 188) days where all 7 children survived including 5 to heart transplant, 1 to recovery, and 1 to conversion to a paracorporeal device. One patient experienced an ischemic stroke on day 53 of device support in the setting of myocardial recovery. One patient required ECMO support for intractable ventricular arrhythmias and was eventually transplanted from paracorporeal biventricular VAD support. The median pump speed was 1600 RPM with power ranging from 1–4 Watts. The median plasma free hemoglobin was 19, 30, 19 and 30 mg/dL at 7, 30, 90 and 180 days or time of explant, respectively. All patients reached the primary feasibility endpoint. Patient-reported outcomes with the device were favorable with respect to participation in a full range of activities. Due to financial issues with the manufacturer, the study was suspended after consent of the eighth patient. The Jarvik 2015 LVAD appears to hold important promise as an implantable continuous flow device for smaller children that may support hospital discharge. The FDA has approved the device to proceed to a 22-subject pivotal trial. Whether this device will survive to commercialization remains unclear because of the financial challenges faced by industry seeking to develop pediatric medical devices. (Supported by NIH/NHLBI HHS Contract N268201200001I, clinicaltrials.gov 02954497). [ABSTRACT FROM AUTHOR]

We report current outcomes in patients supported with the HeartMate 3 (HM3) ventricular assist device in a multicenter learning network. The Advanced Cardiac Therapies Improving Outcomes Network database was queried for HM3 implants between 12/2017 and 5/2022. Clinical characteristics, postimplant course, and adverse events were collected. Patients were stratified according to body surface area (BSA) (<1.4 m2, 1.4-1.8 m2, and >1.8 m2) at device implantation. During the study period, 170 patients were implanted with the HM3 at participating network centers, with median age 15.3 years; 27.1% were female. Median BSA was 1.68 m2; the smallest patient was 0.73 m2 (17.7 kg). Most (71.8%) had a diagnosis of dilated cardiomyopathy. With a median support time of 102.5 days, 61.2% underwent transplantation, 22.9% remained supported on device, 7.6% died, and 2.4% underwent device explantation for recovery; the remainder had transferred to another institution or transitioned to a different device type. The most common adverse events included major bleeding (20.8%) and driveline infection (12.9%); ischemic and hemorrhagic stroke were encountered in 6.5% and 1.2% of patients, respectively. Patients with BSA <1.4 m2 had a higher incidence of infection, renal dysfunction, and ischemic stroke. In this updated cohort of predominantly pediatric patients supported with the HM3 ventricular assist device, outcomes are excellent with <8% mortality on device. Device-related adverse events including stroke, infection, and renal dysfunction were more commonly seen in smaller patients, highlighting opportunities for improvements in care. [ABSTRACT FROM AUTHOR]

Despite recent data suggesting improved outcomes with bivalirudin vs heparin in pediatric Ventricular assist devices (VAD), higher costs remain a barrier. This study quantified trends in bivalirudin use and compared outcomes, resource utilization, and cost-effectiveness associated with bivalirudin vs heparin. Children age 0 to 6 year who received VAD from 2009 to 2021 were identified in Pediatric Health Information System. Bivalirudin use was evaluated using trend analysis and outcomes were compared using Fine-Gray subdistrubtion hazard ratios (SHR). Daily-level hospital costs were compared due to differences in length of stay. Cost-effectiveness was evaluated using incremental cost-effectiveness ratio (ICER). Of 691 pediatric VAD recipients (median age 1 year, IQR 0-2), 304 (44%) received bivalirudin with 90% receiving bivalirudin in 2021 (trend p -value <0.01). Bivalirudin had lower hospital mortality (26% vs 32%; adjusted SHR 0.57, 95% CI 0.40-0.83) driven by lower VAD mortality (20% vs 27%; adjusted SHR 0.46, 95% CI 0.32-0.77) after adjusting for year, age, diagnosis, and center VAD volume. Post-VAD length of stay was longer for bivalirudin than heparin (median 91 vs 64 days, respectively, p < 0.001). Median daily-level costs were lower among bivalirudin (cost ratio 0.87, 95% CI 0.79-0.96) with higher pharmacy costs offset by lower imaging, laboratory, supply, and room/board costs. Estimated ICER for bivalirudin vs heparin was $61,192 per quality-adjusted life year gained with a range of $27,673 to $131,243. Bivalirudin use significantly increased over the past decade and is now used in 90% young pediatric VAD recipients. Bivalirudin was associated with significantly lower hospital mortality and an ICER <$65,000, making it a cost-effective therapy for pediatric VAD recipients. [ABSTRACT FROM AUTHOR]

Cardiac disease results in significant morbidity and mortality in patients with muscular dystrophy (MD). Single centers have reported their ventricular assist device (VAD) experience in specific MDs and in limited numbers. This study sought to describe the outcomes associated with VAD therapy in an unselected population across multiple centers. We examined outcomes of patients with MD and dilated cardiomyopathy implanted with a VAD at Advanced Cardiac Therapies Improving Outcomes Network (ACTION) centers from 9/2012 to 9/2020. A total of 19 VADs were implanted in 18 patients across 12 sites. The majority of patients had dystrophinopathy (66%) and the median age at implant was 17.2 years (range 11.7-29.5). Eleven patients were non-ambulatory (61%) and 6 (33%) were on respiratory support pre-VAD. Five (28%) patients were implanted as a bridge to transplant, 4 of whom survived to transplant. Of 13 patients implanted as bridge to decision or destination therapy, 77% were alive at 1 year and 69% at 2 years. The overall frequencies of positive outcome (transplanted or alive on device) at 1 year and 2 years were 84% and 78%, respectively. Two patients suffered a stroke, 2 developed sepsis, 1 required tracheostomy, and 1 experienced severe right heart failure requiring right-sided VAD. This study demonstrates the potential utility of VAD therapies in patients with muscular dystrophy. Further research is needed to further improve outcomes and better determine which patients may benefit most from VAD therapy in terms of survival and quality of life. [ABSTRACT FROM AUTHOR]

Derangements in liver and renal function often accompany end-stage heart failure. We sought to assess the utility of an objective risk assessment tool, the Model for End-stage Liver Disease eXcluding INR (MELD-XI), to identify pediatric patients at increased risk for adverse outcomes post-ventricular assist device (VAD) implantation. The Pedimacs database was queried for all pediatric patients who underwent VAD implantation from September 19, 2012 to December 31, 2019. Pre-implant and early (1-week) post-implant MELD-XI scores were used to stratify patients into low, intermediate and high score cohorts. Comparison of pre-implant characteristics and post-implant outcomes were conducted across groups. Multiphase parametric hazard modeling was utilized to identify independent predictors of post-implant mortality. A total of 742 patients had a calculable MELD-XI score pre-implant. When stratified by MELD-XI scores pre-implant, patients in the high MELD-XI score cohort (score >13.6) had inferior survival and increased bleeding, renal dysfunction and respiratory failure post-implant compared to intermediate and low score cohorts. Risk factors for mortality post-VAD implantation were: increasing MELD-XI scores (HR 1.1 per 1 unit rise), Pedimacs profile 1 (HR 1.6), congenital heart disease (HR 2.3) and being on a percutaneous VAD (HR 2.7). Importantly, MELD-XI score was a better predictor of post-VAD implant mortality than bilirubin or creatinine alone, neither of which were significant in the final model. Patients with increasing or continued high MELD-XI scores early post-implant had the worst survival. The MELD-XI is an easily calculated score that serves as a promising risk assessment tool in identifying children at risk for poor outcomes post VAD implantation. [ABSTRACT FROM AUTHOR]

• Ventricular assist device (VAD) recipients should be monitored closely for new HLA antibody (Ab) formation. • The majority of pediatric and adult patients had pre-VAD HLA Ab sampling. • The vast majority of pediatric and adult patient had post-VAD HLA Ab sampling. • For more than one third of patients, the HLA lab was unaware of VAD status pretransplant. • Inconsistencies exist in peri-VAD HLA Ab sampling and communication to the HLA laboratory. Ventricular assist devices (VADs) are commonly used as a bridge to transplantation but may yield HLA sensitization. We evaluated the prevalence of HLA antibody (Ab) sampling pre- and post-VAD placement in pediatric and adult patients and notification of VAD status to the HLA laboratory. All pediatric and adult patients who received a first-time VAD between 2005 and 2013 were included in this single-center retrospective review. Data were collected from the University of Alberta Hospital histocompatibility laboratory's information system and a local VAD database. In total, 106 patients were included (40 pediatric, median 3.0 years [interquartile range, 0.3-10.7]; 66 adult, 55.0 years [46.8-61.2]). HLA Ab sampling within 1-month pre-VAD occurred in 70% of pediatric and 79% of adult recipients (P =.215). Testing within 1 month of VAD placement occurred in 89% of pediatric and 67% of adult recipients (P =.012). For those with HLA Ab sampling within 30 days postimplant, notification to the HLA laboratory of VAD status occurred in 19 of 27 (70%) pediatric and 24 of 33 (73%) adult patients (P =.533). Of patients transplanted post VAD with HLA Ab samples collected, 12 of 28 (43%) and 13 of 38 (34%) adult recipients did not have notification of VAD status to the HLA laboratory (P =.322). There were inconsistencies in HLA Ab sampling and communication to the HLA laboratory surrounding VAD placement. Standardization of both HLA Ab assessment frequency after VAD implantation and communication regarding changes in clinical status and the occurrence of key sensitizing events such as VAD placement are imperative as patients await transplantation. [ABSTRACT FROM AUTHOR]

Coronary allograft vasculopathy (CAV) is a leading cause of mortality after heart transplantation (HT) in children. Variation in CAV screening practices may impact detection rates and patient outcomes. Among 50 Pediatric Heart Transplant Society (PHTS) sites from 2001 to 2016, coronary evaluations were classified as angiography or non-invasive testing, and angiograms were designated as routine or symptom based. CAV detection rates stratified by routine vs symptom-based angiograms were calculated. Freedom from CAV and mortality after CAV diagnosis, stratified by study indication, were calculated. A total of 3,442 children had 13,768 coronary evaluations; of these, 97% (n = 13,012) were for routine surveillance, and only 3% (n = 333) were for cause. Over the study period, CAV was detected in 472 patients (14%). Whereas 58% (n = 29) of PHTS sites evaluate by angiography alone, 42% reported supplementing with a non-invasive test, although only 423 non-invasive studies were reported. Angiographic detection of CAV was higher for symptom-based testing than for routine testing (29% vs 4%, p < 0.0001), although routine testing identified a majority of cases (88%; n = 414). The 10-year freedom from CAV was 77% overall. Once CAV is detected, 5-year graft survival was 58%, with lower survival for patients diagnosed after symptoms angiogram than after routine angiogram (30% vs 62%; p < 0.0001). Development of a robust model for CAV risk should allow low-risk patients to undergo less frequent invasive angiography without adverse impact on CAV detection rates or outcomes. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

BACKGROUND There has been increasing use of durable ventricular assist devices (VAD) in children as a bridge to transplantation (BTT). The Berlin Heart investigational device exemption (IDE) trial was the first pediatric VAD trial to demonstrate excellent survival outcomes as a BTT. OBJECTIVES Our aim was to compare the expanded post-transplant outcomes for children enrolled in the Berlin Heart IDE trial to a matched Pediatric Heart Transplant Study (PHTS) cohort not requiring mechanical circulatory support (MCS). SETTING University Hospitals. METHODS This was a retrospective review of linked PHTS and Berlin Heart IDE databases for pediatric (≤18 years) recipients transplanted from 2007-2011. Subjects with <5 years of follow up were excluded. VAD supported patients were matched 1:2 to non-VAD supported controls from the PHTS database. RESULTS Among 109 Berlin Heart IDE study enrollees, 83 were merged with the PHTS database and matched to 166 non-MCS supported patients. There was no difference in diagnosis, status at listing, and age between groups with the expected difference in inotrope use in the non-MCS supported patients. Compared to their matched cohort, there was no statistical difference in 5-year patient survival between VAD and non-VAD patients (81% vs 88%; p = 0.09) nor was there a difference in freedom from rejection or infection. CONCLUSIONS This data suggests that children supported with a Berlin Heart VAD had similar survival, infection and rejection rates compared to those not requiring MCS support. Continued surveillance of the Berlin Heart IDE trial population post heart transplantation is warranted. [ABSTRACT FROM AUTHOR]

Background Children requiring heart transplantation (HTx) for congenital heart disease (CHD) or failing anatomically normal hearts (CMP) face different challenges pre-HTx. We compared the neurocognitive capabilities in pre–school-age children receiving HTx for CHD vs CMP and determined factors predicting outcomes. Methods Data were collected within a prospective multi-provincial project from children who underwent HTx ≤4 years of age between 1999 and 2011. At age 54 ± 3 months, we obtained scores from the Wechsler Preschool and Primary Scales of Intelligence for full-scale intelligence quotient (FSIQ) verbal intelligence quotient (VIQ) and performance intelligence quotient (PIQ), and from the Beery–Buktenica Developmental Test for visual–motor integration (VMI). Possible predictive factors were collected prospectively from transplant listing. Results Of the 76 patients included in the study, 61 survived to assessment, 2 were lost to follow-up and 4 were excluded for genetic disorders or heart–lung transplant. The CHD patients ( n = 32) had significantly more previous surgeries, more severe kidney injuries, more days on ventilator and in intensive care, broader human leukocyte antigen (HLA) sensitization, longer cardipulmonary bypass (CPB) times and higher inotropic scores than CMP patients ( n = 23). Mean IQ scores for the HTx children were below population norms and significantly lower in children with CHD. Intellectual disability (FSIQ <70) was more common in the CHD group ( p = 0.036). The lower VMI in CHD patients approached significance. Lower FSIQ and VMI were independently associated with higher pre-HTx creatinine and lactate, longer stay in intensive care and lower socioeconomic status. Conclusions Children post-HTx showed IQ and VMI scores within the borderline to low-average range, with CHD children ranging significantly lower. Low scores are associated with a more difficult pre- and peri-transplant course. Careful follow-up is required to warrant early detection of deficits and introduction of interventions and supportive measures. [ABSTRACT FROM AUTHOR]

Background Management of mechanical circulatory support in children with congenital heart disease (CHD) is challenging due to physiologic variations and anatomic limitations to device placement. In this study we examine the use of Berlin Heart EXCOR in CHD patients. Methods CHD patients were identified from the EXCOR Pediatric Study data set (2007 to 2010). Mortality and serious adverse events were compared between CHD and non-CHD cohorts, and predictors of poor outcomes in the CHD cohort were identified. Results CHD was present in 29% ( n = 59, 18 with 1-ventricle physiology) of all EXCOR patients ( N = 204). Successful bridge (transplant or wean) was less likely in CHD patients compared with non-CHD patients (48% vs 80%; p < 0.01). Among CHD patients, no neonates, 25% of infants (30 days to 1 year) and 65% of children (>1 year) were successfully bridged. Pre-implant congenital heart surgery (CHS) and extracorporeal membrane oxygenation (ECMO) on the same admission occurred in 60% of children ≤1 year of age (83% of neonates, 50% of infants), with 8% survival. Regardless of age, patients who did not have CHS and ECMO had 61% survival. Smaller pump, pre-implant bilirubin >1.2 mg/dl and renal dysfunction were independently associated with mortality. Conclusions End-organ function at implant reliably predicts adverse outcomes and should be considered when making implant decisions. EXCOR use in neonates and infants with CHD should be approached cautiously. If patients have undergone pre-implant CHS and ECMO, EXCOR support may not provide any survival benefit. EXCOR support in non-infants with CHD is challenging but can be consistently successful with appropriate patient selection. [ABSTRACT FROM AUTHOR]

Background Use of mechanical circulatory support in children has increased as more options have become available. A national account of the use of mechanical support in children and adolescents is essential to understanding outcomes, refining patient selection and improving quality of care. Methods The Pediatric Interagency Registry for Mechanical Circulatory Support (PediMACS) is a National Heart, Lung, and Blood Institute-supported nationwide registry for temporary and durable ventricular assist device (VAD) use in patients <19 years of age. Between the launch in September 2012 and June 2015, 37 hospitals in the USA have enrolled patients. This first report of data from PediMACS analyzed pre-implant patient characteristics, survival using competing outcomes, and adverse events. Results Two hundred pediatric patients underwent 222 durable VAD implants. Patients’ characteristics and outcomes of children supported with a temporary device ( n = 41) were not analyzed in this report. The etiology of heart disease included 146 (73%) patients with cardiomyopathy and 35 (18%) with congenital heart disease. Thirty patients (15%) transitioned from extracorporeal membrane oxygenation (ECMO) and 76 (38%) had previous cardiac surgery. Most patients were Interagency Registry for Mechanically Assisted Circulatory Support (INTERMACS) Level 1 (27%) or Level 2 (56%) at implant, with 13% at Level 3. Of the 200 patients supported with a durable device, 91 (46%) were supported with a pulsatile-flow device and 109 (55%) with a continuous-flow (CF) device. Patient age at first implant included 30 patients (15%) <1 year of age, 37 (19%) 1 to 5 years, 32 (16%) 6 to 10 years and 101 (51%) 10 to 18 years. Patients were supported with left ventricular assist device alone in 161 (81%), biventricular ventricular assist device in 29 (15%), right ventricular assist device in 4 (2.0%) and total artificial heart in 6 (3%), together comprising 783 months of follow-up. The 200 patients receiving primary durable devices had an actuarial survival of 81% at 6 months. Competing risk analysis at 6 months revealed that 58% of patients had been transplanted, 28% were alive on support, 14% had died and 0.6% recovered. In the overall cohort, there were 28 deaths. Reported serious adverse events included infection ( n = 78), bleeding ( n = 68), device malfu n ction ( n = 79) and neurologic dysfunction ( n = 52). Conclusions PediMACS constitutes the largest single data repository with detailed information of pediatric patients implanted with VADs. The first PediMACS report reveals favorable outcomes despite the varying patient characteristics and pump types. However, the rate of adverse events remains high. With further data collection, analysis of patient risk factors critical to improving outcomes will be possible. [ABSTRACT FROM AUTHOR]