Systemic ventricular assist device support in Fontan patients: A report by ACTION.

Background: The size of the Fontan population with end-stage heart failure is growing. In this population, heart transplantation has been the only option. This study sought to investigate the efficacy of ventricular assist device (VAD) support in Fontan patients.

Distinct genetic pathways define pre-malignant versus compensatory clonal hematopoiesis in Shwachman-Diamond syndrome.

To understand the mechanisms that mediate germline genetic leukemia predisposition, we studied the inherited ribosomopathy Shwachman-Diamond syndrome (SDS), a bone marrow failure disorder with high risk of myeloid malignancies at an early age. To define the mechanistic basis of clonal hematopoiesis in SDS, we investigate somatic mutations acquired by patients with SDS followed longitudinally. Here we report that multiple independent somatic hematopoietic clones arise early in life, most commonly harboring heterozygous mutations in EIF6 or TP53.

Berlin Heart EXCOR and ACTION post-approval surveillance study report.

Background: The Berlin Heart EXCOR Pediatric (EXCOR) ventricular assist device (VAD) was introduced in North America nearly 2 decades ago. The EXCOR was approved under Humanitarian Device Exemption status in 2011 and received post-market approval (PMA) in 2017 from Food and Drug Administration. Since the initial approval, the field of pediatric mechanical circulatory support has changed, specifically with regard to available devices, anticoagulation strategies, and the types of patients supported.

Global Leadership in Paediatric and Congenital Cardiac Care: "Coding our way to improved care: an interview with Rodney C. G. Franklin, MBBS, MD, FRCP, FRCPCH".

Dr Rodney Franklin is the focus of our third in a planned series of interviews in Cardiology in the Young entitled, "Global Leadership in Paediatric and Congenital Cardiac Care." Dr Franklin was born in London, England, spending the early part of his childhood in the United States of America before coming back to England. He then attended University College London Medical School and University College Hospital in London, England, graduating in 1979.

MRI surveillance of boys with X-linked adrenoleukodystrophy identified by newborn screening: Meta-analysis and consensus guidelines.

Background: Among boys with X-Linked adrenoleukodystrophy, a subset will develop childhood cerebral adrenoleukodystrophy (CCALD). CCALD is typically lethal without hematopoietic stem cell transplant before or soon after symptom onset. We sought to establish evidence-based guidelines detailing the neuroimaging surveillance of boys with neurologically asymptomatic adrenoleukodystrophy.

Role of Theophylline in Management of Bradycardia Secondary to High Cervical Spinal Cord Injury in a Seven-Year-Old Child: Case Report and a Review of Literature.

High-level cervical spinal cord injury (SCI) frequently leads to the development of severe sinus bradycardia and asystole. Conventionally, owing to their chronotropic effects, medical management has largely relied on the use of atropine and/or infusion of pressors such as epinephrine or dopamine as the first-line treatment. However, for severe symptomatic events refractory to medical therapy, cardiac pacemaker implantation may be required.

Evaluation of time to clearance of high-dose methotrexate using the ARK immunoassay in pediatric hematology/oncology patients: A single-center experience.

A retrospective, single-center, observational study evaluated the difference in time to clearance of high-dose methotrexate between the fluorescence polarization immunoassay (FPIA) and ARK methotrexate assay in pediatric hematology/oncology patients. The post-ARK immunoassay group had an increase in clearance time of 33 h/cycle and 31% increase in cycles with delayed clearance. On posthoc analysis, use of an adjusted clearance threshold of <0.

Survival After Heart Transplant Listing for Infants on Mechanical Circulatory Support.

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.

Specific Class I HLA Supertypes but Not HLA Zygosity or Expression Are Associated with Outcomes following HLA-Matched Allogeneic Hematopoietic Cell Transplant: HLA Supertypes Impact Allogeneic HCT Outcomes.

Maximizing the probability of antigen presentation to T cells through diversity in HLAs can enhance immune responsiveness and translate into improved clinical outcomes, as evidenced by the association of heterozygosity and supertypes at HLA class I loci with improved survival in patients with advanced solid tumors treated with immune checkpoint inhibitors. We investigated the impact of HLA heterozygosity, supertypes, and surface expression on outcomes in adult and pediatric patients with acute myeloid leukemia (AML), myelodysplastic syndrome, acute lymphoblastic leukemia, and non-Hodgkin lymphoma who underwent 8/8 HLA-matched, T cell replete, unrelated, allogeneic hematopoietic cell transplant (HCT) from 2000 to 2015 using patient data reported to the Center for International Blood and Marrow Transplant Research. HLA class I heterozygosity and HLA expression were not associated with overall survival, relapse, transplant-related mortality (TRM), disease-free survival (DFS), and acute graft-versus-host disease following HCT.

ECMO for severe ARDS: systematic review and individual patient data meta-analysis.

Purpose: To assess the effect of venovenous extracorporeal membrane oxygenation (ECMO) compared to conventional management in patients with severe acute respiratory distress syndrome (ARDS).

Methods: We conducted a systematic review and individual patient data meta-analysis of randomised controlled trials (RCTs) performed after Jan 1, 2000 comparing ECMO to conventional management in patients with severe ARDS. The primary outcome was 90-day mortality.

Global Leadership in Paediatric and Congenital Cardiac Care: "Using data to improve outcomes - an interview with Jennifer S. Li, MD, MHS".

Dr. Jennifer Li is the focus of our second in a planned series of interviews in Cardiology in the Young entitled, "Global Leadership in Paediatric and Congenital Cardiac Care". Dr.

Severe COVID-19 After Recent Heart Transplantation Complicated by Allograft Dysfunction.

A 50-year-old male presented with atrial flutter 25 days after heart and kidney transplantation. Rejection was excluded, but he developed severe COVID-19 infection with cardiac allograft dysfunction. Despite continued corticosteroid and tacrolimus therapy, he remained aviremic.

Emphysematous Cystitis Following Bone Marrow Transplant.

Emphysematous cystitis is an exceedingly rare complication of urinary tract infection in children and adults. Characterized by air within the bladder wall, this life-threatening condition most often impacts diabetic women. The inciting pathogen is typically Escherichia coli or Klebsiella pneumoniae, though many other organisms have also been reported.

Global Leadership in Paediatric and Congenital Cardiac Care: "Following the 'Golden Rule' in multicentre collaborations - an interview with Jane W. Newburger, MD".

Dr. Jane Newburger is the focus of our first in a planned series of interviews in Cardiology in the Young entitled, "Global Leadership in Paediatric and Congenital Cardiac Care". Dr.

Potential Risk Factors Associated With Graft Failure of Haploidentical Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease.

Nonmyeloablative (NMA) haploidentical hematopoietic stem cell transplantation for sickle cell disease has significantly increased donor availability for transplant and is increasingly used as curative treatment. The authors describe 3 pediatric patients who rejected grafts after an NMA regimen, previously reported to result in good engraftment rates in the mainly adult population. In this manuscript, potential factors contributing to rejection are described and discussed.

Description of Bivalirudin Use for Anticoagulation in Pediatric Patients on Mechanical Circulatory Support.

Background: Although heparin has previously been the anticoagulant of choice during mechanical circulatory support (MCS), there is a lack of consistency in dose-response in pediatric patients. Bivalirudin offers more consistent dose-response in adults; however, there are limited data for pediatrics use.

Objective: The purpose was to characterize the usage, dosage, and safety profile of bivalirudin when used for pediatric MCS in a tertiary care pediatric hospital.

Risk Factors for Graft-versus-Host Disease in Haploidentical Hematopoietic Cell Transplantation Using Post-Transplant Cyclophosphamide.

Post-transplant cyclophosphamide (PTCy) has significantly increased the successful use of haploidentical donors with a relatively low incidence of graft-versus-host disease (GVHD). Given its increasing use, we sought to determine risk factors for GVHD after haploidentical hematopoietic cell transplantation (haplo-HCT) using PTCy. Data from the Center for International Blood and Marrow Transplant Research on adult patients with acute myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, or chronic myeloid leukemia who underwent PTCy-based haplo-HCT (2013 to 2016) were analyzed and categorized into 4 groups based on myeloablative (MA) or reduced-intensity conditioning (RIC) and bone marrow (BM) or peripheral blood (PB) graft source.

Results of a Randomized, Double-Blinded, Placebo-Controlled, Phase 2.5 Study of Saracatinib (AZD0530), in Patients with Recurrent Osteosarcoma Localized to the Lung.

Purpose: Osteosarcoma is a rare cancer and a third of patients who have completed primary treatment will develop osteosarcoma recurrence. The Src pathway has been implicated in the metastatic behavior of osteosarcoma; about 95% of samples examined express Src or have evidence of downstream activation of this pathway. Saracatinib (AZD0530) is a potent and selective Src kinase inhibitor that was evaluated in adults in Phase 1 studies.

Prenatal Treatment of Congenital Cytomegalovirus With Valganciclovir: A Case Report.

Cytomegalovirus (CMV) is the most common congenital infection and infectious cause of fetal anomaly and neurologic injury. However, treatment strategies for congenital CMV (cCMV) infection during pregnancy remain elusive. We report a case of hydrops fetalis secondary to cCMV infection with minimal sequelae after maternal and subsequent neonatal treatment with valganciclovir.

Continuous Versus Bolus Gastric Feeding in Children Receiving Mechanical Ventilation: A Systematic Review.

Background: Nutrition guidelines recommend enteral nutrition in the form of gastric feedings for critically ill children and acknowledge a lack of evidence describing an optimal method for providing these feedings.

Objective: To determine the state of the science regarding the efficacy of bolus (intermittent) or continuous gastric feedings to improve nutrition delivery in critically ill children receiving mechanical ventilation.

Methods: Five hundred seventy-nine abstracts met the inclusion criteria and were screened by 2 reviewers according to prespecified criteria.

An Evaluation of Risperidone Dosing for Pediatric Delirium in Children Less Than or Equal to 2 Years of Age.

Risperidone dosing and safety data are limited in patients ≤2 years of age. To describe the dosing strategies, safety, and tolerability of risperidone in infants ≤2 years of age. An institutional review board-approved retrospective study was conducted in a 24-bed pediatric intensive care unit at an academic medical center in patients ≤2 years of age receiving risperidone for the management of ICU delirium.

Prospective clinical testing and experimental validation of the Pediatric Sepsis Biomarker Risk Model.

Sepsis remains a major public health problem with no major therapeutic advances over the last several decades. The clinical and biological heterogeneity of sepsis have limited success of potential new therapies. Accordingly, there is considerable interest in developing a precision medicine approach to inform more rational development, testing, and targeting of new therapies.

Assessing Nursing and Pediatric Resident Understanding of Delirium in the Pediatric Intensive Care Unit.

Delirium is a common disease process in the pediatric critical care unit, yet practices for screening and prevention vary drastically between institutions. The authors hypothesized that surveying pediatric residents and nurses who care for patients in the intensive care setting would expose misunderstandings about delirium. They brought to light common incorrect beliefs that benzodiazepines are appropriate therapy for delirium and that children who are delirious will not have memories of the experience.

Developments in Pediatric Ventricular Assist Device Support.

Mechanical support devices have revolutionized the management of circulatory failure. This has been met by a dramatic increase in ventricular assist device (VAD) utilization in children over the last two decades. Previous wide gaps in experience and knowledge have significantly narrowed in the recent years.