<p><b>PURPOSE: </b>With advances in mechanical circulatory support and orthotopic heart transplants (OHT) remaining a limited resource, there has been a dramatic increase in Ventricular Assist Device (VAD) implantation. There is minimal data comparing emergency department (ED) resource utilization and outcomes between these populations. We examined national estimates of VAD and OHT-related ED visits and evaluated admissions, resource utilization, and mortality.</p><p><b>METHODS: </b>This study is an epidemiological analysis comparing national estimates of ED visit-level data from the 2006-2014 Nationwide Emergency Department Sample (NEDS) in patients with VADs vs OHT, identified using ICD-9 codes. The primary outcome was death; secondary outcomes included median inflation-adjusted charge and hospital admission. We tested the hypothesis that resource utilization and mortality are higher in ED visits for VAD patients compared to OHT patients.</p><p><b>RESULTS: </b>17,356 VAD-related ED, and 138,133 OHT-related visits were identified. Patients with VADs were more likely to be male (74% vs 70%, p=0.001) and ≥ 65 yo (39% vs 38%, p=0.0004). VADs were more likely to have a primary diagnosis of bleeding (25% vs 2%) and less likely to have acute respiratory disease (6% vs 20%, p<0.0001 for both). VAD-related ED visits had higher rates of inpatient admission or transfer (73% vs 57%) and a higher mortality rate (4.7% vs 1.8%) than patients with OHT (p <0.0001 for all). Moreover, VAD related ED visits had higher median inflation-adjusted charges [$23,862 (IQR $7,129-$58,265) vs $11,364 (IQR $3,001-$31,694)] (p<0.0001).</p><p><b>CONCLUSION: </b>Patients with VADs presenting to the ED represent a population with greater morbidity, mortality and resource utilization compared to OHT. A more developed understanding of those factors that drive mortality and resource use is imperative for improving outcomes in this high-risk population.</p>

<p><b>PURPOSE: </b>Currently there are no-FDA approved immunosuppressants specific to pediatric heart transplantation (HT). In recent years, everolimus (EVL) has emerged as an alternative to tacrolimus (TAC) as a primary immunosuppressant to prevent rejection that may also prevent kidney and coronary disease. However, the two regimens have never been evaluated systematically.</p><p><b>METHODS: </b>The TEAMMATE Trial (IND 127980) is designed to evaluate the safety and efficacy of EVL and low-dose (LD-TAC) compared to standard-therapy TAC and mycophenolate mofetil (MMF). The study design and rationale are reviewed in light of challenges inherent in rare disease research.</p><p><b>RESULTS: </b>The TEAMMATE trial is the first multicenter randomized clinical trial (RCT) in pediatric HT. The primary purpose is to evaluate the risk-benefit profile of the two regimens to prevent major adverse transplant events (MATE), and to support FDA approval of 1 or both regimens for pediatric HT. Children <21 years at HT will be randomized (1:1 ratio) at 6 mo. post-HT to either regimen for 30 months (Figure). Children with recurrent rejection or a GFR <60 ml/min/1.73m2 are excluded. The primary efficacy hypothesis is that compared to TAC/MMF, EVL/LD-TAC is more effective in preventing 3 MATEs: cellular rejection, CKD and CAV. The primary safety hypothesis is that EVL/LD-TAC does not have a higher cumulative burden of 6 MATE (AMR, infection, and PTLD + the 3 above). The primary endpoint is the MATE Score, a surrogate endpoint reflecting the frequency and severity of MATEs and validated against graft loss. The study will enroll 210 patients across 26 sites and is powered to demonstrate superior efficacy of EVL/LDTAC. The trial is projected to be completed in 2022.</p><p><b>CONCLUSION: </b>The TEAMMATE trial is the first RCT in pediatric HT. It is anticipated that the study will provide important information about the safety and effectiveness of EVL and TAC and provide valuable lessons into the design and conduct of future trials in pediatric HT.</p>

<p><b>PURPOSE: </b>Data related to the epidemiology and resource utilization of ventricular assist device (VAD) related emergency department (ED) visits are limited. However, an improved understanding of the burden of VADs on the healthcare system is critical to designing interventions that improve outcomes. We examined national estimates of VAD-related ED visits and described medical complexity, admissions, resource utilization, and mortality.</p><p><b>METHODS: </b>This study utilizes data from ED encounter-level data via the 2006-2014 Nationwide Emergency Department Sample (NEDS). ICD-9 codes were used to identify patient-encounters with VADs. Demographic and clinical factors are reported via descriptive statistics. The primary outcome was death; secondary outcomes included median inflation-adjusted charge and hospital admission.</p><p><b>RESULTS: </b>From 2006 to 2014, over 900 million ED visits were evaluated, of which 17,356 (.002%) VAD-related ED visits were identified, for a median of 1,028 (SD 1,489) visits per year. Most VAD ED encounters were represented by patients that were male (74%), ≥ 45 years old (73%), and living in an urban environment (85%). 73% of VAD related ED visits resulted in inpatient admission and the median inflation-adjusted charge was $23,862 (IQR 7,129; 58,265) per visit. Mortality, either in the ED or during an associated admission, was 4.7%. The most common primary diagnoses were cardiac (32%) followed by bleeding (25%) and infection (18%). More than half of the encounters were with patients with ≥1 chronic medical condition, with diabetes (34%) and hypertension (31%) the most prevalent.</p><p><b>CONCLUSION: </b>This is the first study to use national level data to describe the clinical characteristics and outcomes of patients with VADs who present to the ED. With nearly 1 in 20 VAD ED encounters resulting in death, strategies to reduce mortality are urgently needed.</p>

<p><b>PURPOSE: </b>Heart transplantation (HT) in children consumes substantial health care resources. However, little is known regarding the financing of pediatric HT in the US and whether there have been changes in the nature of pediatric HT financing over time.</p><p><b>METHODS: </b>The United Network for Organ Sharing thoracic organ transplant database was queried for all HTs performed in patients <18 years of age between 1994 and 2018. Primary payer status at the time of HT was identified for all HT recipients and classified as either private, Medicaid, or other. Trends over time were analyzed using linear regression. Payer status was similarly examined for patients at the time of waiting list addition.</p><p><b>RESULTS: </b>During the study period, 8289 HTs were performed in patients <18 years of age, with primary payer information available for 98.9% of the cohort. The annual number of pediatric HTs increased from 196 in 1994 to 465 in 2018. There were significant changes in primary payer over time. In 1994, private insurance and Medicaid covered 49.0% and 40.3% of recipients, respectively, while by 2018, the dominant proportion changed such that private insurance and Medicaid covered 38.9% and 51.4% of recipients, respectively (P<0.0001) (Figure 1). Similar trends were observed for primary payer status at the time of waiting list addition (P<0.0001).</p><p><b>CONCLUSION: </b>Currently, pediatric HT in the US is funded predominantly by Medicaid. In recent years, the proportion of patients with private insurance has fallen with a commensurate increase in Medicaid as primary payer. These findings have implications in view of recent threats to Medicaid funding nationally. Further study is necessary to better understand the causes and impacts of these temporal changes.</p>