Pediatric Myelodysplastic Syndrome.

Context.—: Myelodysplastic syndromes (MDSs) are rare in children and have unique clinical manifestations and implications.

Objective.

Clinical Impact of a Standardized Risk-Stratified Thromboprophylaxis Protocol for Multisystem Inflammatory Syndrome in Children.

Objective: To evaluate the clinical impact of an institutional thromboprophylaxis protocol in patients with multisystem inflammatory syndrome in children (MIS-C), who are at increased risk for thromboembolism (TE).

Study Design: We conducted a single-center retrospective cohort study of children less than 18 years between March 2020 and December 2021. Eligible patients were confirmed with MIS-C and were managed with a standardized multidisciplinary treatment approach that included a thromboprophylaxis protocol to guide and unify clinical practice.

Medich Giant Platelet Syndrome: An Evolving Qualitative and Quantitative Platelet Disorder.

Qualitative platelet disorders remain rare and varied. We describe here 2 additional patients with giant platelets, thrombocytopenia, deficiency in alpha granules and the presence of membranous inclusions within the cytoplasm. Collectively known as Medich syndrome, we further elucidated structural and clinical features of this rare syndrome.

Cerebral Sinus Venous Thrombosis in Infants after Surgery for Congenital Heart Disease.

Objective: To determine the prevalence of and risk factors for cerebral sinus venous thrombosis (CSVT) in neonates undergoing congenital heart disease (CHD) repair.

Study Design: Neonates who had CHD repair with cardiopulmonary bypass and postoperative brain magnetic resonance imaging (MRI) between 2013 and 2019 at a single tertiary care center were identified from institutional databases. Demographic, clinical, and surgical data were abstracted from these databases and from the medical record; 278 neonates with CHD had cardiopulmonary bypass, 184 of whom had a postoperative brain MRI.

Hospital-Associated Venous Thromboembolism in a Pediatric Cardiac ICU: A Multivariable Predictive Algorithm to Identify Children at High Risk.

Objectives: Critically ill children with cardiac disease are at significant risk for hospital-associated venous thromboembolism, which is associated with increased morbidity, hospital length of stay, and cost. Currently, there are no widely accepted guidelines for prevention of hospital-associated venous thromboembolism in pediatrics. We aimed to develop a predictive algorithm for identifying critically ill children with cardiac disease who are at increased risk for hospital-associated venous thromboembolism as a first step to reducing hospital-associated venous thromboembolism in this population.

Characteristics and outcomes of osteomyelitis in children with sickle cell disease: A 10-year single-center experience.

Background: Patients with sickle cell disease (SCD) are at increased risk for osteomyelitis (OM). Diagnosis of OM in SCD is challenging as the clinical presentation is similar to a vasoocclusive crisis (VOC) with no diagnostic gold standard. We report characteristics and outcomes of OM in SCD patients treated at our center over 10-year period.

Targeted Inhibition of the Epidermal Growth Factor Receptor and Mammalian Target of Rapamycin Signaling Pathways in Olmsted Syndrome.

Importance: Olmsted syndrome is a rare and disabling genodermatosis for which no successful treatment is currently available.

Objective: To evaluate the clinical response to the mammalian target of rapamycin (mTOR) inhibitor sirolimus and/or the epidermal growth factor receptor (EGFR) inhibitor erlotinib among patients with Olmsted syndrome.

Design, Setting, And Participants: This case series focused on 4 children with treatment-refractory Olmsted syndrome.

Atlanto-occipital ligament calcification: a novel imaging finding in pediatric rotational vertebral artery occlusion.

We describe a 2-year-old girl with bow hunter syndrome complicated by vertebral artery dissection and multiple ischemic infarcts. Pediatric bow hunter syndrome is a rare and likely under-recognized disorder. Interestingly, our patient had atlanto-occipital ligament calcification on CT scan, an imaging finding that has not been reported in association with bow hunter syndrome and one that might help increase recognition of this dynamic disorder of the posterior circulation.

Cardiopulmonary Bypass Reduces Early Thrombosis of Systemic-to-Pulmonary Artery Shunts.

Background: Shunt thrombosis is a significant cause of morbidity and mortality after systemic-to-pulmonary artery shunt (SPS) placement. Concurrent procedures with placement of SPS may require cardiopulmonary bypass (CPB). Cardiopulmonary bypass is known to cause bleeding and platelet dysfunction in infants, which may protect from early shunt thrombosis.

Catheter-Related Venous Thrombosis in Hospitalized Pediatric Patients with Inflammatory Bowel Disease: Incidence, Characteristics, and Role of Anticoagulant Thromboprophylaxis with Enoxaparin.

Objective: To describe the incidence and characteristics of central venous catheter (CVC)-related thrombosis in hospitalized pediatric patients with active inflammatory bowel disease (IBD) and report the potential usefulness of anticoagulant thromboprophylaxis (AT).

Study Design: We conducted a retrospective study of patients who were admitted to our children's hospital in the last 2 years with active IBD and required a CVC and identified all patients with an objectively confirmed symptomatic CVC-related thrombosis. To assess the usefulness of a recently implemented institutional AT protocol, we compared the frequency of CVC-related thrombosis, nadir hemoglobin, and red blood cell transfusion requirements in patients who received AT with those who did not during the study period.

IV Versus Subcutaneous Enoxaparin in Critically Ill Infants and Children: Comparison of Dosing, Anticoagulation Quality, Efficacy, and Safety Outcomes.

Objective: Subcutaneous enoxaparin is the mainstay anticoagulant in critically ill pediatric patients although it poses several challenges in this patient population. Enoxaparin infused IV over 30 minutes represents an attractive alternative, but there is limited experience with this route of administration in children. In this study, we assess dosing, anticoagulation quality, safety, and clinical efficacy of IV enoxaparin compared to subcutaneous enoxaparin in critically ill infants and children.

Acquired von Willebrand Syndrome: An Under-Recognized Cause of Major Bleeding in the Cardiac Intensive Care Unit.

Background: Acquired von Willebrand syndrome (AvWS) in the setting of congenital heart disease is an under-recognized cause of bleeding in the pediatric cardiac critical care unit.

Methods: Fourteen patients diagnosed with AvWS admitted to the cardiac intensive care unit at the Children's National Health System between December 2009 and September 2015 were identified with subsequent chart review and case analysis.

Results: Of the 14 patients included in this study, 4 patients were on ventricular-assist devices, 6 patients were on extracorporeal membrane oxygenation, and 4 were patients with congenital heart disease not receiving any mechanical circulatory support.

Transcatheter Treatment of Thrombosis in the Single Ventricle Pathway: An Institutional Experience.

Background: Shunt or conduit thrombosis in a single ventricle circuit is a life-threatening complication that requires prompt treatment to rapidly restore shunt/conduit patency. Transcatheter interventions represent an attractive alternative to systemic thrombolysis or open surgical procedures. We report our center's experience with catheter-based approaches in patients with palliated single ventricle who present with shunt/conduit thrombosis.

Monitoring the harm associated with use of anticoagulants in pediatric populations through trigger-based automated adverse-event detection.

Background: The safety profile of anticoagulants, which are being used with increasing frequency in pediatric populations, is not well studied. Automatic triggers built into electronic health record systems (EHR) have been shown to be an effective way to monitor for and identify medication errors. Anticoagulant-associated adverse events were examined through the use of an anticoagulant trigger panel.

Isolated midbrain ischemic infarct in association with hyperlipoproteinemia (a): a report of 2 adolescent patients.

Arterial ischemic strokes (AIS) localized solely to the midbrain are extremely uncommon in the pediatric population. Elevated lipoprotein (a), which promotes atherosclerosis and a prothrombotic state, has been associated with increased risk of AIS in children and adults. Here we describe a 17-year-old boy and a 15-year-old girl who presented with internuclear ophthalmoplegia secondary to an isolated midbrain AIS.

Coagulopathy in newborns with hypoxic ischemic encephalopathy (HIE) treated with therapeutic hypothermia: a retrospective case-control study.

Background: Newborns with hypoxic ischemic encephalopathy (HIE) are at risk for coagulopathy due to systemic oxygen deprivation. Additionally, therapeutic hypothermia (TH) slows enzymatic activity of the coagulation cascade, leading to constitutive prolongation of routinely assessed coagulation studies. The level of laboratory abnormality that predicts bleeding is unclear, leading to varying transfusion therapy practices.

Acquired von Willebrand syndrome in a child following Berlin Heart EXCOR Pediatric Ventricular Assist Device implantation: case report and concise literature review.

The development of acquired von Willebrand syndrome (AVWS) after placement of a pulsatile-flow left ventricular assist device (LVAD) is rare and only recently recognized. We report the case of a young infant who was diagnosed with ventricular assist device (VAD)-related AVWS following implantation of a Berlin Heart EXCOR Pediatric Ventricular Assist Device (Berlin Heart Inc., The Woodlands, Texas, USA) for treatment of severe heart failure.

Massive transfusion in children and neonates.

Resuscitation of children and neonates with severe or refractory bleeding due to surgery or trauma often requires massive transfusion (MT). Findings from recent studies have led to a better understanding of the complex pathophysiology in massive haemorrhage and the effects of MT on haemostasis. Current management of the massively bleeding adult patient has evolved over the past few decades, shifting to early transfusion of products in a balanced ratio as part of MT protocols (MTPs).

Interstitial deletion of 11q-implicating the KIRREL3 gene in the neurocognitive delay associated with Jacobsen syndrome.

Jacobsen syndrome (JS) is a rare contiguous gene disorder characterized by a deletion within the distal part of the long arm of chromosome 11 ranging in size from 7 to 20 Mb. The clinical findings include characteristic dysmorphic features, growth and psychomotor delays and developmental anomalies involving the brain, eyes, heart, kidneys, immune, hematologic, endocrine, and gastrointestinal systems. The majority of cases are due to a terminal deletion of 11q; however interstitial deletions have also been reported.

Pulmonary embolism in children.

Pulmonary embolism (PE) has long been described in children. Nevertheless, most of the algorithms applied to patients within this age range, from diagnosis to therapy, have been adapted from adult protocols. This article reviews the progresses that occurred to PE in children placing them in historical perspective with the key events relevant to PE in adults.

Acquired cytochrome C oxidase impairment in apheresis platelets during storage: a possible mechanism for depletion of metabolic adenosine triphosphate.

Background: Intracellular adenosine triphosphate (ATP) levels decline significantly during storage of platelet (PLT) products, in part due to PLT degranulation. However, metabolic ATP stores also become depleted during storage through an unclear mechanism. Since both anaerobic glycolysis and oxidative phosphorylation are important for PLT ATP production, it is possible that the reduction in metabolic ATP reflects impaired oxidative phosphorylation.