Publications by authors named "Elfriede Pahl"
Background: Extending survival after heart transplant (HT) is of paramount importance for childhood recipients of HT. Acute rejection is a significant event, and biopsy remains the most specific means for distinguishing between cellular (ACR) and antibody-mediated rejection (AMR).
Methods: All children in the Pediatric Heart Transplant Society Registry who underwent HT between January 2015 and June 2022 and had ≥1 rejection episode were included.
Background: Pediatric dilated cardiomyopathy often leads to death or cardiac transplantation. We sought to determine whether changes in left ventricular (LV) end-diastolic dimension (LVEDD), LV end-diastolic posterior wall thickness, and LV fractional shortening (LVFS) over time may help predict adverse outcomes.
Methods And Results: We studied children up to 18 years old with dilated cardiomyopathy, enrolled between 1990 and 2009 in the Pediatric Cardiomyopathy Registry.
Background: Myocardial fibrosis, as diagnosed on cardiac magnetic resonance imaging (cMRI) by late gadolinium enhancement (LGE), is associated with adverse outcomes in adults with hypertrophic cardiomyopathy (HCM), but its prevalence and magnitude in children with HCM have not been established. We investigated: (1) the prevalence and extent of myocardial fibrosis as detected by LGE cMRI; (2) the agreement between echocardiographic and cMRI measurements of cardiac structure; and (3) whether serum concentrations of N-terminal pro hormone B-type natriuretic peptide (NT-proBNP) and cardiac troponin-T are associated with cMRI measurements.
Methods: A cross-section of children with HCM from 9 tertiary-care pediatric heart centers in the U.
Article Synopsis
- * The study involves children under 21 years old, randomizing them at 6 months post-transplant for a 30-month follow-up, aiming to determine which immunosuppression regimen is more effective at preventing acute cellular rejection, chronic kidney disease, and cardiac allograft vasculopathy.
- * The trial's primary endpoint is the MATE score, which measures the frequency and severity of major adverse transplant events, and is designed
Background: Despite ubiquitous exposure to sensitizing events, most Fontan PLE patients have low panel reactive antibodies (PRA). To assess whether they are at risk for donor-specific antibody (DSA) memory response following heart transplantation (HT) when their PLE resolves, DSA profiles, incidence of rejection, and graft outcomes in Fontan recipients with and without PLE were compared.
Methods: Patient characteristics, appearance of newly detected DSA (nDSA), and graft outcomes were compared between patients with and without PLE using Wilcoxon rank-sum and Chi-squared tests.
Objectives: Donor-specific cell-free DNA shows promise as a noninvasive marker for allograft rejection, but as yet has not been validated in both adult and pediatric recipients. The study objective was to validate donor fraction cell-free DNA as a noninvasive test to assess for risk of acute cellular rejection and antibody-mediated rejection after heart transplantation in pediatric and adult recipients.
Methods: Pediatric and adult heart transplant recipients were enrolled from 7 participating sites and followed for 12 months or more with plasma samples collected immediately before all endomyocardial biopsies.
Background: Ideal "cardiovascular health" (CVH)-optimal diet, exercise, nonsmoking, BMI, BP, lipids, and glucose-is associated with healthy longevity in adults. Pediatric heart transplant (HT) patients may be at risk for suboptimal CVH.
Methods: Single-center retrospective study of HT patients 2003-2014 who survived 1 year post-transplant.
Background: Clinical rejection (CR) defined as decision to treat clinically suspected rejection with change in immunotherapy based on clinical presentation with or without diagnostic biopsy findings is an important part of care in heart transplantation. We sought to assess the utility of donor fraction cell-free DNA (DF cfDNA) in CR and the utility of serial DF cfDNA in CR patients in predicting outcomes of clinical interest.
Methods: Patients with heart transplantation were enrolled in two sequential, multi-center, prospective observational studies.
Background: Obesity and dyslipidemia afflict children of all ages. We explored the prevalence of obesity and dyslipidemia in pediatric heart transplant (HT) recipients and its effects on cardiac allograft vasculopathy (CAV) and survival.
Methods: This study included primary HT recipients (≤18 years) transplanted between 01/1996 and 12/2018 included in the Pediatric Heart Transplant Society database.
To understand the genetic contribution to primary pediatric cardiomyopathy, we performed exome sequencing in a large cohort of 528 children with cardiomyopathy. Using clinical interpretation guidelines and targeting genes implicated in cardiomyopathy, we identified a genetic cause in 32% of affected individuals. Cardiomyopathy sub-phenotypes differed by ancestry, age at diagnosis, and family history.
View Article and Find Full Text PDFBackground Pediatric dilated cardiomyopathy (DCM) is a well-known clinical entity; however, phenotype-genotype correlations are inadequately described. Our objective was to provide genotype associations with life-threatening cardiac outcomes in pediatric DCM probands. Methods and Results We performed a retrospective review of children with DCM at a large pediatric referral center (2007-2016), excluding syndromic, chemotherapy-induced, and congenital heart disease causes.
View Article and Find Full Text PDFBackground: 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.
Methods: 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.
There are little data on postheart transplant (HT) outcomes for pediatric patients that were supported to HT with biventricular assist device (BiVAD). The United Network for Organ Sharing database was queried for patients <18 years old at time of HT between January 2005 and March 2018, excluding patients bridged with total artificial hearts and right ventricular assist device (VAD). Of 4,904 pediatric HT recipients, patients were grouped by no VAD support (3,934; 80.
View Article and Find Full Text PDFBackground: Cell-free DNA is an emerging biomarker. While donor fraction may detect graft events in heart transplant recipients, the prognostic value of total nuclear cell-free DNA (ncfDNA) itself is largely unexplored.
Objective: Explore the relationship between ncfDNA and clinical events in heart transplant recipients.
Background: Challenges exist with heterotaxy due to the complexity of heart disease, abnormal venous connections, and infection risks. This study aims to understand heart transplant outcomes for children with heterotaxy.
Methods: All children with congenital heart disease listed for transplant from 1993 to 2018 were included.
Article Synopsis
- Pediatric cardiomyopathy is a serious heart condition in children with genetic diversity, and there's a need for consistent genetic testing across practices due to significant variations in testing rates.
- A study involving 152 children revealed that 41% had a family history of cardiomyopathy, and 48% of those who had prior testing received positive results.
- The research indicated that genetic testing can uncover the causes of cardiomyopathy in many children, suggesting routine testing may improve diagnosis and management.
Background Infants with heart failure remain at significant risk for wait list mortality, despite mechanical circulatory support (MCS). It is unclear if the outcomes are influenced by modality of support or underlying diagnosis. We sought to compare the outcomes of infants <10 kg, focusing on modality of support and underlying diagnosis.
View Article and Find Full Text PDFBackground: Minimal data exist on clinical decision-making in VAD implantation in pediatrics. This study aims to identify areas of consensus/variability among pediatric VAD physicians in determining eligibility and factors that guide decision-making.
Methods: An 88-item survey with clinical vignettes was sent to 132 pediatric HT cardiologists and surgeons at 37 centers.
Background: 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.
Methods: 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.
Background Coronary artery aneurysms (CAAs) may occur after Kawasaki disease (KD) and lead to important morbidity and mortality. As CAA in patients with KD are rare and heterogeneous lesions, prognostication and risk stratification are difficult. We sought to derive the cumulative risk and associated factors for cardiovascular complications in patients with CAAs after KD.
View Article and Find Full Text PDFBackground: The substantial risk of thrombosis in large coronary artery aneurysms (CAAs) (maximum z-score ≥ 10) after Kawasaki disease (KD) mandates effective thromboprophylaxis. We sought to determine the effectiveness of anticoagulation (low-molecular-weight heparin [LMWH] or warfarin) for thromboprophylaxis in large CAAs.
Methods: Data from 383 patients enrolled in the International KD Registry (IKDR) were used.
Background: Endomyocardial biopsy (EMB) is the current standard for rejection surveillance in heart transplant recipients. The quantification of donor-specific cell-free DNA (cfDNA) may be an appropriate biomarker for non-invasive rejection surveillance. A multicenter prospective blinded study (DNA-Based Transplant Rejection Test, DTRT) investigated the value of donor fraction (DF), defined as the ratio of cfDNA specific to the transplanted organ to the total amount of cfDNA present in a blood sample.
View Article and Find Full Text PDFBackground: The use of ventricular assist device (VAD) in children has increased, but the decision of left VAD (LVAD) vs biventricular support remains a challenge. Children who undergo LVAD placement are at risk for right ventricular failure (RHF), but the incidence has not been described.
Methods: Analysis was performed for patients <18 years old who underwent durable LVAD placement within the Pedimacs registry (September 19, 2012-February 28, 2017), excluding single ventricle morphology and temporary devices.
Background: The VANISH trial (Valsartan for Attenuating Disease Evolution in Early Sarcomeric Hypertrophic Cardiomyopathy) targeted young sarcomeric gene mutation carriers with early-stage hypertrophic cardiomyopathy (HCM) to test whether valsartan can modify disease progression. We describe the baseline characteristics of the VANISH cohort and compare to previous trials evaluating angiotensin receptor blockers.
Methods: Applying a randomized, double-blinded, placebo-controlled design, 178 participants with nonobstructive HCM (age, 23.
Objectives: The time course for hemodynamic normalization after pediatric heart transplantation has not been well characterized. We hypothesized that patients with a single ventricle would normalize later than those with dilated cardiomyopathy. Establishing the expected course based on the underlying pathophysiology will allow identification of patients who are outliers, requiring further investigation.
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