Duration of anticoagulation for venous thromboembolism in pediatric patients: Evaluation of the Duration of Therapy for Thrombosis in Children (Kids-DOTT) trial outcomes at 2 years.

Background: The Multicenter Evaluation of the Duration of Therapy for Thrombosis in Children multinational, randomized clinical trial revealed noninferiority of a 6-week vs 3-month duration of anticoagulation for the treatment of provoked venous thromboembolism (VTE) in patients <21 years old in regard to net clinical benefit at 1 year.

Objectives: To evaluate noninferiority at 2 years.

Methods: Patients whose repeat imaging 6 weeks after VTE diagnosis did not show complete veno-occlusion were randomized to discontinue anticoagulation vs receive a total 3-month course and followed for 2 years for the occurrence of symptomatic recurrent VTE (efficacy outcome) and clinically relevant bleeding (safety outcome).

Hydroxyurea to prevent brain injury in children with sickle cell disease (HU Prevent)-A randomized, placebo-controlled phase II feasibility/pilot study.

Central nervous system (CNS) injury is common in sickle cell disease (SCD) and occurs early in life. Hydroxyurea is safe and efficacious for treatment of SCD, but high-quality evidence from randomized trials to estimate its neuroprotective effect is scant. HU Prevent was a randomized (1:1), double-blind, phase II feasibility/pilot trial of dose-escalated hydroxyurea vs.

Electrochemical Activation of Fe-LiF Conversion Cathodes in Thin-Film Solid-State Batteries.

Transition metal fluoride (TMF) conversion-type cathodes promise up to 4 times higher gravimetric energy densities compared to those of common intercalation-type cathodes. However, TMF cathodes demonstrate sluggish kinetics, poor efficiencies, and incompatibility with many liquid electrolytes. In this work, coevaporated heterostructured iron and lithium fluoride (Fe-LiF) cathodes are investigated in thin-film solid-state batteries with a LiPON electrolyte and a lithium metal anode.

Influence of Au, Pt, and C Seed Layers on Lithium Nucleation Dynamics for Anode-Free Solid-State Batteries.

In the concept of anode-free lithium-ion batteries, cells are manufactured with a bare anode current collector where the lithium metal anode is electrochemically formed from the lithium-containing cathode during the first charge cycle. While this concept has many attractive aspects from a manufacturing and energy density standpoint, stable plating and stripping remain challenging. We have investigated gold, platinum, and amorphous carbon as seed layers placed between the copper current collector and the lithium phosphorus oxynitride thin-film solid electrolyte.

Monolithically-stacked thin-film solid-state batteries.

The power capability of Li-ion batteries has become increasingly limiting for the electrification of transport on land and in the air. The specific power of Li-ion batteries is restricted to a few thousand W kg due to the required cathode thickness of a few tens of micrometers. We present a design of monolithically-stacked thin-film cells that has the potential to increase the power ten-fold.

Current developmental screening practices in young children with sickle cell disease.

Despite recent developmental screening guidelines, rates of neurodevelopmental disorders (NDDs) remain lower than expected in children with sickle cell disease (SCD). A retrospective chart review identified 276 eligible patients; 214 charts were available for developmental screening and 207 charts for autism-specific screening. Developmental surveillance/screening was conducted in 70% of charts and autism-specific screening in 19% of charts.

Brain Oxygen Extraction and Metabolism in Pediatric Patients With Sickle Cell Disease: Comparison of Four Calibration Models.

Sickle cell disease (SCD) is an inherited hemoglobinopathy with an increased risk of neurological complications. Due to anemia and other factors related to the underlying hemoglobinopathy, cerebral blood flow (CBF) increases as compensation; however, the nature of alterations in oxygen extraction fraction (OEF) and cerebral metabolic rate of oxygen (CMRO) in SCD remains controversial, largely attributed to the different calibration models. In addition, limited studies have been done to investigate oxygen metabolism in pediatric patients.

Effect of Anticoagulant Therapy for 6 Weeks vs 3 Months on Recurrence and Bleeding Events in Patients Younger Than 21 Years of Age With Provoked Venous Thromboembolism: The Kids-DOTT Randomized Clinical Trial.

Importance: Among patients younger than 21 years of age, the optimal duration of anticoagulant therapy for venous thromboembolism is unknown.

Objective: To test the hypothesis that a 6-week duration of anticoagulant therapy for provoked venous thromboembolism is noninferior to a conventional 3-month therapy duration in patients younger than 21 years of age.

Design, Setting, And Participants: Randomized clinical trial involving 417 patients younger than 21 years of age with acute, provoked venous thromboembolism enrolled at 42 centers in 5 countries from 2008-2021.

Imaging Blood-Brain Barrier Permeability Through MRI in Pediatric Sickle Cell Disease: A Feasibility Study.

Background: Blood-brain barrier (BBB) disruption may lead to endothelium dysfunction and inflammation in sickle cell disease (SCD). However, abnormalities of BBB in SCD, especially in pediatric patients for whom contrast agent administration less than optimal, have not been fully characterized.

Purpose: To examine BBB permeability to water in a group of pediatric SCD participants using a non-invasive magnetic resonance imaging technique.

Co-Occurrence of Neurodevelopmental Disorders in Pediatric Sickle Cell Disease.

Objective: The objective of this study is to retrospectively determine the co-occurrence, associated characteristics, and risk factors for neurodevelopmental disorders (NDD) in a pediatric sickle cell disease (SCD) clinic population.

Method: We investigated the co-occurrence and features of NDD in pediatric SCD through a retrospective cohort study conducted between July 2017 and January 2019. The participants were patients with SCD younger than 18 years of age identified from our institutions' clinic rosters and medical records databases.

Perioperative Management of Pediatric Patients with Moyamoya Arteriopathy.

Pediatric patients with moyamoya arteriopathy are at high risk for developing new onset transient or permanent neurologic deficits secondary to cerebral hypoperfusion, particularly in the perioperative period. It is therefore essential to carefully manage these patients in a multidisciplinary, coordinated effort to reduce the risk of new permanent neurologic deficits. However, little has been published on perioperative management of pediatric patients with moyamoya, particularly in the early postoperative period during intensive care unit admission.

Proteomic discovery in sickle cell disease: Elevated neurogranin levels in children with sickle cell disease.

Purpose: Sickle cell disease (SCD) is an inherited hemoglobinopathy that causes stroke and silent cerebral infarct (SCI). Our aim was to identify markers of brain injury in SCD.

Experimental Design: Plasma proteomes were analyzed using a sequential separation approach of hemoglobin (Hb) and top abundant plasma protein depletion, followed by reverse phase separation of intact proteins, trypsin digestion, and tandem mass spectrometry.

Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial.

Importance: Although effective agents are available to prevent painful vaso-occlusive episodes of sickle cell disease (SCD), there are no disease-modifying therapies for ongoing painful vaso-occlusive episodes; treatment remains supportive. A previous phase 3 trial of poloxamer 188 reported shortened duration of painful vaso-occlusive episodes in SCD, particularly in children and participants treated with hydroxyurea.

Objective: To reassess the efficacy of poloxamer 188 for vaso-occlusive episodes.

Association of Antibiotic Choice With Hospital Length of Stay and Risk Factors for Readmission in Patients With Sickle Cell Disease and Acute Chest Syndrome: An Observational Cohort Study.

Study Objective: We determine the association between use of specific cephalosporins and macrolides and hospital length of stay in patients with sickle cell disease (SCD) who are admitted with acute chest syndrome, and determine treatment risk factors for acute chest syndrome-related 30-day readmission.

Methods: Patients admitted to 48 US hospitals within the Pediatric Health Information System between January 2008 and December 2016 with associated International Classification of Diseases, Ninth Revision (ICD-9) or ICD-10 diagnoses of SCD and acute chest syndrome were included. Primary outcomes were hospital length of stay and acute chest syndrome-related and all-cause 30-day readmission.

Brain-derived neurotrophic factor levels in pediatric sickle cell disease.

Background: Children with sickle cell disease (SCD) have an increased risk of neurological complications, particularly stroke and silent cerebral infarction (SCI). Brain-derived neurotrophic factor (BDNF) is a nerve growth factor associated with neuronal survival, synaptic plasticity, elevated transcranial Doppler (TCD) velocities and increased risk of stroke in patients with SCD. The objective of this study was to analyze plasma BDNF protein levels in children with SCD participating in the Silent Cerebral Infarct Transfusion Multi-Center Clinical Trial (SIT Trial), comparing plasma samples of children with SCD and SCI to plasma samples from children with SCD without SCI, as well as healthy pediatric control participants.

Male reproductive health in cystic fibrosis.

The impact of cystic fibrosis (CF) on male reproductive health is profound. The vast majority of men with CF are infertile due to obstructive azoospermia. Multiple factors associated with CF contribute to an increased prevalence of testosterone deficiency, which adversely affects muscle mass, bone density, and quality of life.

Quantification of whole-brain oxygenation extraction fraction and cerebral metabolic rate of oxygen consumption in adults with sickle cell anemia using individual T -based oxygenation calibrations.

Purpose: To evaluate different T -oxygenation calibrations for estimating venous oxygenation in people with sickle cell anemia (SCA).

Methods: Blood T values were measured at 3 T in the internal jugular veins of 12 healthy volunteers and 11 SCA participants with no history of stroke, recent transfusion, or renal impairment. T -oxygenation relationships of both sickled and normal blood samples were calibrated individually and compared with values generated from published models.

Correlates of successful transition in young adults with sickle cell disease.

The transition period from pediatric care to adult care for patients with sickle cell disease (SCD) is associated with increased mortality and morbidity. Identification of risk factors for unsuccessful transition may aid in developing strategies to improve the transition process and health outcomes in this population. We examined factors associated with unsuccessful transition from pediatric to adult care for patients with SCD at the Johns Hopkins Hospital.