BackgroundA significant number of pediatric heart transplant recipients and their families experi... more BackgroundA significant number of pediatric heart transplant recipients and their families experience post‐traumatic stress symptoms following transplantation, which can impact recipient behavioral and medical health outcomes. Preventive behavioral health interventions may improve outcomes, especially if interventions can be delivered at a distance to decrease barriers to mental health care. This pilot study examined the acceptability and accessibility of an evidence‐informed resilience training program delivered using a video telehealth platform. A secondary aim was to assess the preliminary efficacy of the intervention on recipient behavioral health outcomes, perceived barriers to recipient medication adherence, parent behavioral health outcomes, and family functioning.MethodsSeventeen heart transplant recipients (8–18 years old) and their families were recruited and randomly assigned to a treatment as usual (n = 8) or an intervention group (n = 9). Baseline assessment data collec...
The Journal of Heart and Lung Transplantation, 2017
Results: Twenty-five patients (M age = 14.7, SD age = 2.3) and 20 caregivers completed the web-ba... more Results: Twenty-five patients (M age = 14.7, SD age = 2.3) and 20 caregivers completed the web-based assessment and consultation model. Youth selfreports indicated that 24% (n= 6) had clinically significant emotional and behavioral difficulties, and 20% (n= 5) had impaired prosocial behavior. Notably, parent reports of youth behavior indicated that 45% (n= 9) had clinically significant total difficulties. Of eighteen caregiver self-reports, 11% (n= 2) screened at clinically significant risk for depression, and 39% (n= 7) at risk for poor family functioning. The FOCUS consultation model shows promise for integrating family-centered behavioral health prevention and care approaches within fast-paced outpatient pediatric settings. FOCUS allows the pediatric team to identify and respond to patients' social and psychological obstacles in a timely way. The FOCUS model was well-received by the medical team.
The Journal of Heart and Lung Transplantation, 2012
The purpose of this study was to investigate the use of two CircuLite micro-pumps for full bivent... more The purpose of this study was to investigate the use of two CircuLite micro-pumps for full biventricular assist in sheep. We therefore examined right and left sided hemodynamic parameters over a range of right and left sided pump speeds. Methods and Materials: Five juvenile sheep (mean-weight 43kg) were implanted with CircuLite pumps in the RV and the LV through a median sternotomy. The RVAD outflow graft was anastomosed end-to-side to the pulmonary artery, and the LVAD outflow to the ascending aorta. After surgical implantation of both pumps we induced ventricular fibrillation and hemodynamic changes were measured using nine different levels of RVAD pump speed (from 20-28k rpm at 1k increments) while the speed of the LVAD was set constant at 24k, then at 26k and finally at 28k rpm. Results: Average results from the 5 sheep with LVAD speed fixed at 24K are shown in the Figure . At any given LVAD speed, both RVAD and LVAD flow increased identically as RVAD speed was increased. Results were similar with LVAD set at the higher speeds. At the highest LVAD and RVAD speeds, flow averaged 3.1Ϯ0.7 L/min, and pressures in the right atrium, pulmonary artery, left atrium and aorta averaged 2.2Ϯ3.7, 24.4Ϯ6.5, 22.4Ϯ5.5, and 56.6Ϯ8.5 mmHg, respectively. Conclusions: Biventricular assist with the two CircuLite pumps is feasible and able to provide full hemodynamic support in sheep. This approach holds promise for providing biventricular partial support in humans and, in particular, for full support in small adults and children.
The Journal of Heart and Lung Transplantation, 2012
Purpose: This study validated and characterized the suitability of transcutaneous energy transfer... more Purpose: This study validated and characterized the suitability of transcutaneous energy transfer (TET) to power a novel low-power intra-aortic micro-axial entrainment pump for long-term partial circulatory support. During long-term use, indwelling power leads represent a significant infection risk. A TET system eliminates this risk by transferring power across the skin through inductive coupling. During the study, key design questions were answered and operating requirements and performance parameters were characterized. Methods and Materials: The TET system comprises a Class-E power amplifier with a self-oscillating feedback network and a two-way telemetry system. The system autotunes transfer frequency and power output to optimize efficiency and minimize power consumption. Output current and voltage of the TET system were measured over a range of distance and coil orientations. Major system components are shown in Fig 1 .
The Journal of Heart and Lung Transplantation, 2015
Purpose: Cardiac Allograft Hypertrophy (CAH) develops in up to 80% of patients as early as six mo... more Purpose: Cardiac Allograft Hypertrophy (CAH) develops in up to 80% of patients as early as six months post-transplant, and is associated with decreased, decreased survival, increased myocardial size, extracellular fibrosis and cardiac wall thickening. It is not known if pathways described in non-transplant cardiac hypertrophy play a role in CAH. Methods: We performed full genome analysis in 10 endomyocardial biopsies from 5 patients who underwent heart transplantation. Biopsies were taken at week 4 and 20 post-transplant. We analyzed cardiac hypertrophy pathways within epithelial to mesenchymal transition (EMT), Collagen, Angiotensin, fetal gene and IL6 dependent hypertrophy pathways. Gene expression at 20 weeks was correlated with LV mass measured by echo, myocyte size by H&E staining, acute cellular rejection, C4D deposition, and interstitial fibrosis by Trichrome Mason staining. Results: 8/11 EMT pathway genes were downregulated in most patients. COL1A2, COL3A1, and COL5A1 genes were upregulated with more than 2 fold increase, 8/12 angiotensin pathway genes were upregulated. 4/7 NFAT/GATA4 genes were upregulated in most patients. Results were inconsistent for IL6 dependent pathway. Echo LV mass at week 20 had a significant correlation with the expression of SNAI1, (r= .92; p= 0.026), Col3A1 (r= 0.88, p= 0.046), MAPK9 (r= 0.89, p= 0.038); Myocyte size with MMP2 (r= 0.82, p= 0.046). ACR had a significant correlation with only IL-6 (r= 0.91,p= 0.029) and NFAT C4 (r= 0.92,p= 0.026), C4D deposition had a significant correlation with MMP-9 (r= 0.97, P= 0.004), AGTRAP (r= 0.92, p= 0.023), IL-6R (0.096, p= 0.008) and STAT 4 (r= 0.98,p= 0.003) genes. Early CAH could be caused by increased collagen gene expression and by activation of the angiotensin dependent hypertrophy pathway but not the IL6 dependent pathway. NFAT/GATA4 fetal genome pathways are also upregulated similar to non-transplanted cardiac hypertrophy mechanism.
Among neonatal cardiomyopathies, primary endocardial fibroelastosis (pEFE) remains a mysterious d... more Among neonatal cardiomyopathies, primary endocardial fibroelastosis (pEFE) remains a mysterious disease of the endomyocardium that is poorly genetically characterized, affecting 1/5000 live births and accounting for 25% of the entire pediatric dilated cardiomyopathy (DCM) with a devastating course and grave prognosis. To investigate the potential genetic contribution to pEFE, we performed integrative genomic analysis, using whole exome sequencing (WES) and RNA-seq in a female infant with confirmed pathological diagnosis of pEFE. Within regions of homozygosity in the proband genome, WES analysis revealed novel parent-transmitted homozygous mutations affecting three genes with known roles in cilia assembly or function. Among them, a novel homozygous variant [c.1943delA] of uncertain significance in ALMS1 was prioritized for functional genomic and mechanistic analysis. Loss of function mutations of ALMS1 have been implicated in Alstrom syndrome (AS) [OMIM 203800], a rare recessive cili...
Journal of Heart and Lung Transplantation, Apr 1, 2014
Purpose: Outcomes after listing or heart transplant have been well described in children; however... more Purpose: Outcomes after listing or heart transplant have been well described in children; however, little is known about the outcomes of patients after the initial referral for heart transplantation. Methods: All patients referred to our pediatric heart transplant program between 2004 and 2013 were evaluated. Indications for heart transplantation were consistent with AHA guidelines. Demographics and clinical data were recorded. Competing outcomes were assessed for the study population. Results: 278 patients were evaluated. Ten had an unknown outcome (i.e. lost to follow-up or relocated) and were excluded from analysis. The average age was 6.5 ± 6.8 years and 49.3% were female. The most common diagnoses were congenital heart disease (N= 149, 108 single ventricle) and dilated cardiomyopathy (N= 89). Mechanical circulatory support was being utilized in 31 (11%) patients and 82 (29%) were INTERMACS class 1-2 (Class breakdown: 1, N= 35; 2, N= 47; 3, 71; 4, N= 83; 5, N= 14; 6, N= 5, 7, N= 3) at time of referral. After initial evaluation 86 (30.9%) were listed for HT and 17 (6.4%) were felt not to be HT candidates. For the majority (165, 59.4%) the decision to list for HT was deferred to pursue alternative medical and/ or surgical therapies. Of those who were deferred, 56 (33.9%), 87 (52.7%), and 95 (62.1%) were ultimately listed for HT at 1, 6, and 12 months after initial referral. A logistic regression analysis demonstrated that older patients (OR 1.10 [1.05-1.15], p< 0.0001), those with lower INTERMACS class at referral (OR 1.43 [1.15-1.79], p= 0.001) were more likely while those with congenital heart disease (OR 0.56 [0.32-0.95], p= 0.033) were less likely to be deferred. The waitlist mortality did not differ between those listed immediately versus those who were deferred after initial referral. At time of last follow-up 31 (18.0%) patients continued to be deferred with a median follow up of 19 ± 47.6 weeks with 12 (7%) being deferred for over 1 year. Conclusion: Referral for pediatric HT most often leads to initiation of other therapies and deferring listing for HT. Initiation of alternative therapies at initial HT referral generally leads to delay, rather than avoidance, of HT listing but without any increased risk of waitlist mortality. Patients referred for HT in whom alternative therapies are initiated should be followed closely for deterioration after initial referral.
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation, 1997
The major limiting factor to successful heart transplantation in infants is the limited supply of... more The major limiting factor to successful heart transplantation in infants is the limited supply of donors. To examine the impact of donor limitations on survival after listing, a multiinstitutional study was designed to identify risk factors for death while waiting and for longer interval to transplantation. Between January 1 and December 31, 1993, 118 infants 6 months of age or younger (86 younger than 29 days) were listed for heart transplantation from 21 institutions. The primary diagnosis was hypoplastic left-sided heart syndrome (HLHS) in 70 (59%), other congenital defects in 32 (27%), cardiomyopathy or myocarditis in 13 (11%), and other diagnoses in 3. Among the 48 patients without HLHS, 32 (67%) required inotropic, mechanical, or prostaglandin support, whereas 16 (33%) did not. At 6 months after listing, only 6% remained on the list awaiting transplantation, 59% underwent transplantation. 31% died while waiting, and 4% were removed from the list. The greatest mortality rate be...
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation, 1997
After listing for pediatric heart transplantation, at any point in time one of the following poss... more After listing for pediatric heart transplantation, at any point in time one of the following possibilities could have occurred; death, transplantation, removal from the list because of clinical improvement, or continuing to wait. In the setting of those competing outcomes, the Kaplan-Meier estimate portrays the time-relatedness of an event while ignoring the effect of the other possible outcomes. The competing outcomes method, however, depicts the time relatedness of an event while solving for all possible events simultaneously. The competing outcomes method may potentially provide more accurate information regarding the actual proportion of patients experience an outcome after listing.
Fluid restriction and diuretic management are mainstays in the postoperative management of cardia... more Fluid restriction and diuretic management are mainstays in the postoperative management of cardiac patients, at risk of volume overload and its deleterious effects on primary cardiac function and multi-organ systems. The importance of fluid homeostasis is further emphasized among orthotopic heart transplant recipients (OHT). We sought to investigate the relationship between postoperative volume overload, mortality, and allograft dysfunction among pediatric OHT recipients within 1-year of transplantation. This is a retrospective cohort study from a single pediatric OHT center. Children under 21 years undergoing cardiac transplantation between 2010 and 2018 were included. Cumulative fluid overload (cFO) was assessed as percent fluid accumulation adjusted for preoperative body weight. Greater than 10% cFO defined those with postoperative cFO and a comparison of postoperative cFO vs. no postoperative cFO (< 5%) is reported. 102 pediatric OHT recipients were included. Early cFO at 72 ...
Intensive Care of the Adult with Congenital Heart Disease, 2019
As of 2010 there were approximately 2.4 million people in the United States living with congenita... more As of 2010 there were approximately 2.4 million people in the United States living with congenital heart disease of which 1.4 million are adults (Gilboa SM, Devine OJ, Kucik JE, Oster ME, Riehle-Colarusso T, Nembhard WN, Xu P, Correa A, Jenkins K, Marelli AJ, Circulation. 134:101–9, 2016). With the advancement in prenatal detection, early surgical intervention, and improved medical management, more children with congenital heart disease are living into adulthood. While there has been significant advancement in palliative and corrective surgical repairs of congenital heart disease, congestive heart failure still remains a significant cause for morbidity and mortality in adults with congenital heart disease. As a result, 10–20% of adults with congenital heart disease may require heart transplantation, and they account for approximately 3% of all adult heart transplants (Goldberg SW, Fisher SA, Wehman B, Mehra MR, The Journal of Heart and Lung Transplantation. 33:873–7, 2014). Although...
Background Patients with single‐ventricle physiology who undergo the Fontan procedure are at risk... more Background Patients with single‐ventricle physiology who undergo the Fontan procedure are at risk for thrombotic events associated with significant morbidity and mortality. The UNIVERSE Study evaluated the efficacy and safety of a novel liquid rivaroxaban formulation, using a body weight–adjusted dosing regimen, versus acetylsalicylic acid (ASA) in children post‐Fontan. Methods and Results The UNIVERSE Study was a randomized, multicenter, 2‐part, open‐label study of rivaroxaban, in children who had undergone a Fontan procedure, to evaluate its dosing regimen, safety, and efficacy. Part A was the single‐arm part of the study that determined the pharmacokinetics/pharmacodynamics and safety of rivaroxaban in 12 participants before proceeding to part B, whereby 100 participants were randomized 2:1 to open‐label rivaroxaban versus ASA. The study period was 12 months. A total of 112 participants were enrolled across 35 sites in 10 countries. In part B, for safety outcomes, major bleeding ...
Pulmonary arterial hypertension (PAH) is a rare and progressive disorder. Current treatment in th... more Pulmonary arterial hypertension (PAH) is a rare and progressive disorder. Current treatment in the pediatric population includes phosphodiesterase 5 inhibitors (PDE-5i), endothelin receptor antagonists (ERA), and both inhaled and intravenous prostacyclin pathway agonists. As of December 22, 2015 the first oral prostacyclin pathway agonist, selexipag (Uptravi Ò), was FDA approved in the US. In this case series, we discuss our single-center experience using selexipag in a pediatric population, composed of both patients with idiopathic PAH, and patients with congenital heart disease and PAH.
ASAIO journal (American Society for Artificial Internal Organs : 1992), Jan 11, 2018
Infections in adult ventricular assist device patients increase subsequent mortality and stroke r... more Infections in adult ventricular assist device patients increase subsequent mortality and stroke risk. Less is known about outcomes after infections in younger patients, where diabetes and obesity, risk factors associated with poor outcomes, are less prevalent. The purpose of this study was to examine outcomes after infections in adolescents and young adults with continuous-flow left ventricular assist devices (VAD) bridged to transplant. From Pediatric Interagency Registry for Mechanically Assisted Circulatory Support and Interagency Registry for Mechanical Circulatory Support registries, we identified patients aged 12-29 years with continuous-flow VADs implanted as bridged to transplant from September 2012 to March 2016. The primary predictor variable was first reported infection. The primary outcome was death on VAD support; secondary outcome was clinical stroke. Kaplan-Meier and Cox proportional hazard methods were used to compare outcomes between patients before or without infec...
BackgroundA significant number of pediatric heart transplant recipients and their families experi... more BackgroundA significant number of pediatric heart transplant recipients and their families experience post‐traumatic stress symptoms following transplantation, which can impact recipient behavioral and medical health outcomes. Preventive behavioral health interventions may improve outcomes, especially if interventions can be delivered at a distance to decrease barriers to mental health care. This pilot study examined the acceptability and accessibility of an evidence‐informed resilience training program delivered using a video telehealth platform. A secondary aim was to assess the preliminary efficacy of the intervention on recipient behavioral health outcomes, perceived barriers to recipient medication adherence, parent behavioral health outcomes, and family functioning.MethodsSeventeen heart transplant recipients (8–18 years old) and their families were recruited and randomly assigned to a treatment as usual (n = 8) or an intervention group (n = 9). Baseline assessment data collec...
The Journal of Heart and Lung Transplantation, 2017
Results: Twenty-five patients (M age = 14.7, SD age = 2.3) and 20 caregivers completed the web-ba... more Results: Twenty-five patients (M age = 14.7, SD age = 2.3) and 20 caregivers completed the web-based assessment and consultation model. Youth selfreports indicated that 24% (n= 6) had clinically significant emotional and behavioral difficulties, and 20% (n= 5) had impaired prosocial behavior. Notably, parent reports of youth behavior indicated that 45% (n= 9) had clinically significant total difficulties. Of eighteen caregiver self-reports, 11% (n= 2) screened at clinically significant risk for depression, and 39% (n= 7) at risk for poor family functioning. The FOCUS consultation model shows promise for integrating family-centered behavioral health prevention and care approaches within fast-paced outpatient pediatric settings. FOCUS allows the pediatric team to identify and respond to patients' social and psychological obstacles in a timely way. The FOCUS model was well-received by the medical team.
The Journal of Heart and Lung Transplantation, 2012
The purpose of this study was to investigate the use of two CircuLite micro-pumps for full bivent... more The purpose of this study was to investigate the use of two CircuLite micro-pumps for full biventricular assist in sheep. We therefore examined right and left sided hemodynamic parameters over a range of right and left sided pump speeds. Methods and Materials: Five juvenile sheep (mean-weight 43kg) were implanted with CircuLite pumps in the RV and the LV through a median sternotomy. The RVAD outflow graft was anastomosed end-to-side to the pulmonary artery, and the LVAD outflow to the ascending aorta. After surgical implantation of both pumps we induced ventricular fibrillation and hemodynamic changes were measured using nine different levels of RVAD pump speed (from 20-28k rpm at 1k increments) while the speed of the LVAD was set constant at 24k, then at 26k and finally at 28k rpm. Results: Average results from the 5 sheep with LVAD speed fixed at 24K are shown in the Figure . At any given LVAD speed, both RVAD and LVAD flow increased identically as RVAD speed was increased. Results were similar with LVAD set at the higher speeds. At the highest LVAD and RVAD speeds, flow averaged 3.1Ϯ0.7 L/min, and pressures in the right atrium, pulmonary artery, left atrium and aorta averaged 2.2Ϯ3.7, 24.4Ϯ6.5, 22.4Ϯ5.5, and 56.6Ϯ8.5 mmHg, respectively. Conclusions: Biventricular assist with the two CircuLite pumps is feasible and able to provide full hemodynamic support in sheep. This approach holds promise for providing biventricular partial support in humans and, in particular, for full support in small adults and children.
The Journal of Heart and Lung Transplantation, 2012
Purpose: This study validated and characterized the suitability of transcutaneous energy transfer... more Purpose: This study validated and characterized the suitability of transcutaneous energy transfer (TET) to power a novel low-power intra-aortic micro-axial entrainment pump for long-term partial circulatory support. During long-term use, indwelling power leads represent a significant infection risk. A TET system eliminates this risk by transferring power across the skin through inductive coupling. During the study, key design questions were answered and operating requirements and performance parameters were characterized. Methods and Materials: The TET system comprises a Class-E power amplifier with a self-oscillating feedback network and a two-way telemetry system. The system autotunes transfer frequency and power output to optimize efficiency and minimize power consumption. Output current and voltage of the TET system were measured over a range of distance and coil orientations. Major system components are shown in Fig 1 .
The Journal of Heart and Lung Transplantation, 2015
Purpose: Cardiac Allograft Hypertrophy (CAH) develops in up to 80% of patients as early as six mo... more Purpose: Cardiac Allograft Hypertrophy (CAH) develops in up to 80% of patients as early as six months post-transplant, and is associated with decreased, decreased survival, increased myocardial size, extracellular fibrosis and cardiac wall thickening. It is not known if pathways described in non-transplant cardiac hypertrophy play a role in CAH. Methods: We performed full genome analysis in 10 endomyocardial biopsies from 5 patients who underwent heart transplantation. Biopsies were taken at week 4 and 20 post-transplant. We analyzed cardiac hypertrophy pathways within epithelial to mesenchymal transition (EMT), Collagen, Angiotensin, fetal gene and IL6 dependent hypertrophy pathways. Gene expression at 20 weeks was correlated with LV mass measured by echo, myocyte size by H&E staining, acute cellular rejection, C4D deposition, and interstitial fibrosis by Trichrome Mason staining. Results: 8/11 EMT pathway genes were downregulated in most patients. COL1A2, COL3A1, and COL5A1 genes were upregulated with more than 2 fold increase, 8/12 angiotensin pathway genes were upregulated. 4/7 NFAT/GATA4 genes were upregulated in most patients. Results were inconsistent for IL6 dependent pathway. Echo LV mass at week 20 had a significant correlation with the expression of SNAI1, (r= .92; p= 0.026), Col3A1 (r= 0.88, p= 0.046), MAPK9 (r= 0.89, p= 0.038); Myocyte size with MMP2 (r= 0.82, p= 0.046). ACR had a significant correlation with only IL-6 (r= 0.91,p= 0.029) and NFAT C4 (r= 0.92,p= 0.026), C4D deposition had a significant correlation with MMP-9 (r= 0.97, P= 0.004), AGTRAP (r= 0.92, p= 0.023), IL-6R (0.096, p= 0.008) and STAT 4 (r= 0.98,p= 0.003) genes. Early CAH could be caused by increased collagen gene expression and by activation of the angiotensin dependent hypertrophy pathway but not the IL6 dependent pathway. NFAT/GATA4 fetal genome pathways are also upregulated similar to non-transplanted cardiac hypertrophy mechanism.
Among neonatal cardiomyopathies, primary endocardial fibroelastosis (pEFE) remains a mysterious d... more Among neonatal cardiomyopathies, primary endocardial fibroelastosis (pEFE) remains a mysterious disease of the endomyocardium that is poorly genetically characterized, affecting 1/5000 live births and accounting for 25% of the entire pediatric dilated cardiomyopathy (DCM) with a devastating course and grave prognosis. To investigate the potential genetic contribution to pEFE, we performed integrative genomic analysis, using whole exome sequencing (WES) and RNA-seq in a female infant with confirmed pathological diagnosis of pEFE. Within regions of homozygosity in the proband genome, WES analysis revealed novel parent-transmitted homozygous mutations affecting three genes with known roles in cilia assembly or function. Among them, a novel homozygous variant [c.1943delA] of uncertain significance in ALMS1 was prioritized for functional genomic and mechanistic analysis. Loss of function mutations of ALMS1 have been implicated in Alstrom syndrome (AS) [OMIM 203800], a rare recessive cili...
Journal of Heart and Lung Transplantation, Apr 1, 2014
Purpose: Outcomes after listing or heart transplant have been well described in children; however... more Purpose: Outcomes after listing or heart transplant have been well described in children; however, little is known about the outcomes of patients after the initial referral for heart transplantation. Methods: All patients referred to our pediatric heart transplant program between 2004 and 2013 were evaluated. Indications for heart transplantation were consistent with AHA guidelines. Demographics and clinical data were recorded. Competing outcomes were assessed for the study population. Results: 278 patients were evaluated. Ten had an unknown outcome (i.e. lost to follow-up or relocated) and were excluded from analysis. The average age was 6.5 ± 6.8 years and 49.3% were female. The most common diagnoses were congenital heart disease (N= 149, 108 single ventricle) and dilated cardiomyopathy (N= 89). Mechanical circulatory support was being utilized in 31 (11%) patients and 82 (29%) were INTERMACS class 1-2 (Class breakdown: 1, N= 35; 2, N= 47; 3, 71; 4, N= 83; 5, N= 14; 6, N= 5, 7, N= 3) at time of referral. After initial evaluation 86 (30.9%) were listed for HT and 17 (6.4%) were felt not to be HT candidates. For the majority (165, 59.4%) the decision to list for HT was deferred to pursue alternative medical and/ or surgical therapies. Of those who were deferred, 56 (33.9%), 87 (52.7%), and 95 (62.1%) were ultimately listed for HT at 1, 6, and 12 months after initial referral. A logistic regression analysis demonstrated that older patients (OR 1.10 [1.05-1.15], p< 0.0001), those with lower INTERMACS class at referral (OR 1.43 [1.15-1.79], p= 0.001) were more likely while those with congenital heart disease (OR 0.56 [0.32-0.95], p= 0.033) were less likely to be deferred. The waitlist mortality did not differ between those listed immediately versus those who were deferred after initial referral. At time of last follow-up 31 (18.0%) patients continued to be deferred with a median follow up of 19 ± 47.6 weeks with 12 (7%) being deferred for over 1 year. Conclusion: Referral for pediatric HT most often leads to initiation of other therapies and deferring listing for HT. Initiation of alternative therapies at initial HT referral generally leads to delay, rather than avoidance, of HT listing but without any increased risk of waitlist mortality. Patients referred for HT in whom alternative therapies are initiated should be followed closely for deterioration after initial referral.
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation, 1997
The major limiting factor to successful heart transplantation in infants is the limited supply of... more The major limiting factor to successful heart transplantation in infants is the limited supply of donors. To examine the impact of donor limitations on survival after listing, a multiinstitutional study was designed to identify risk factors for death while waiting and for longer interval to transplantation. Between January 1 and December 31, 1993, 118 infants 6 months of age or younger (86 younger than 29 days) were listed for heart transplantation from 21 institutions. The primary diagnosis was hypoplastic left-sided heart syndrome (HLHS) in 70 (59%), other congenital defects in 32 (27%), cardiomyopathy or myocarditis in 13 (11%), and other diagnoses in 3. Among the 48 patients without HLHS, 32 (67%) required inotropic, mechanical, or prostaglandin support, whereas 16 (33%) did not. At 6 months after listing, only 6% remained on the list awaiting transplantation, 59% underwent transplantation. 31% died while waiting, and 4% were removed from the list. The greatest mortality rate be...
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation, 1997
After listing for pediatric heart transplantation, at any point in time one of the following poss... more After listing for pediatric heart transplantation, at any point in time one of the following possibilities could have occurred; death, transplantation, removal from the list because of clinical improvement, or continuing to wait. In the setting of those competing outcomes, the Kaplan-Meier estimate portrays the time-relatedness of an event while ignoring the effect of the other possible outcomes. The competing outcomes method, however, depicts the time relatedness of an event while solving for all possible events simultaneously. The competing outcomes method may potentially provide more accurate information regarding the actual proportion of patients experience an outcome after listing.
Fluid restriction and diuretic management are mainstays in the postoperative management of cardia... more Fluid restriction and diuretic management are mainstays in the postoperative management of cardiac patients, at risk of volume overload and its deleterious effects on primary cardiac function and multi-organ systems. The importance of fluid homeostasis is further emphasized among orthotopic heart transplant recipients (OHT). We sought to investigate the relationship between postoperative volume overload, mortality, and allograft dysfunction among pediatric OHT recipients within 1-year of transplantation. This is a retrospective cohort study from a single pediatric OHT center. Children under 21 years undergoing cardiac transplantation between 2010 and 2018 were included. Cumulative fluid overload (cFO) was assessed as percent fluid accumulation adjusted for preoperative body weight. Greater than 10% cFO defined those with postoperative cFO and a comparison of postoperative cFO vs. no postoperative cFO (< 5%) is reported. 102 pediatric OHT recipients were included. Early cFO at 72 ...
Intensive Care of the Adult with Congenital Heart Disease, 2019
As of 2010 there were approximately 2.4 million people in the United States living with congenita... more As of 2010 there were approximately 2.4 million people in the United States living with congenital heart disease of which 1.4 million are adults (Gilboa SM, Devine OJ, Kucik JE, Oster ME, Riehle-Colarusso T, Nembhard WN, Xu P, Correa A, Jenkins K, Marelli AJ, Circulation. 134:101–9, 2016). With the advancement in prenatal detection, early surgical intervention, and improved medical management, more children with congenital heart disease are living into adulthood. While there has been significant advancement in palliative and corrective surgical repairs of congenital heart disease, congestive heart failure still remains a significant cause for morbidity and mortality in adults with congenital heart disease. As a result, 10–20% of adults with congenital heart disease may require heart transplantation, and they account for approximately 3% of all adult heart transplants (Goldberg SW, Fisher SA, Wehman B, Mehra MR, The Journal of Heart and Lung Transplantation. 33:873–7, 2014). Although...
Background Patients with single‐ventricle physiology who undergo the Fontan procedure are at risk... more Background Patients with single‐ventricle physiology who undergo the Fontan procedure are at risk for thrombotic events associated with significant morbidity and mortality. The UNIVERSE Study evaluated the efficacy and safety of a novel liquid rivaroxaban formulation, using a body weight–adjusted dosing regimen, versus acetylsalicylic acid (ASA) in children post‐Fontan. Methods and Results The UNIVERSE Study was a randomized, multicenter, 2‐part, open‐label study of rivaroxaban, in children who had undergone a Fontan procedure, to evaluate its dosing regimen, safety, and efficacy. Part A was the single‐arm part of the study that determined the pharmacokinetics/pharmacodynamics and safety of rivaroxaban in 12 participants before proceeding to part B, whereby 100 participants were randomized 2:1 to open‐label rivaroxaban versus ASA. The study period was 12 months. A total of 112 participants were enrolled across 35 sites in 10 countries. In part B, for safety outcomes, major bleeding ...
Pulmonary arterial hypertension (PAH) is a rare and progressive disorder. Current treatment in th... more Pulmonary arterial hypertension (PAH) is a rare and progressive disorder. Current treatment in the pediatric population includes phosphodiesterase 5 inhibitors (PDE-5i), endothelin receptor antagonists (ERA), and both inhaled and intravenous prostacyclin pathway agonists. As of December 22, 2015 the first oral prostacyclin pathway agonist, selexipag (Uptravi Ò), was FDA approved in the US. In this case series, we discuss our single-center experience using selexipag in a pediatric population, composed of both patients with idiopathic PAH, and patients with congenital heart disease and PAH.
ASAIO journal (American Society for Artificial Internal Organs : 1992), Jan 11, 2018
Infections in adult ventricular assist device patients increase subsequent mortality and stroke r... more Infections in adult ventricular assist device patients increase subsequent mortality and stroke risk. Less is known about outcomes after infections in younger patients, where diabetes and obesity, risk factors associated with poor outcomes, are less prevalent. The purpose of this study was to examine outcomes after infections in adolescents and young adults with continuous-flow left ventricular assist devices (VAD) bridged to transplant. From Pediatric Interagency Registry for Mechanically Assisted Circulatory Support and Interagency Registry for Mechanical Circulatory Support registries, we identified patients aged 12-29 years with continuous-flow VADs implanted as bridged to transplant from September 2012 to March 2016. The primary predictor variable was first reported infection. The primary outcome was death on VAD support; secondary outcome was clinical stroke. Kaplan-Meier and Cox proportional hazard methods were used to compare outcomes between patients before or without infec...
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Papers by Juan Alejos