Percutaneous Endoscopic Gastrostomy Enhances Interstage Growth in Infants With Hypoplastic Left Heart Syndrome.

Objectives: Infants with hypoplastic left heart syndrome undergoing staged palliation commonly experience chronic growth failure and malnutrition. Greater patient weight at stage 2 palliation (Glenn) is thought to be associated with improved perioperative outcomes. We aimed to compare weight for age z score and interstage growth velocity in children with and without a percutaneous endoscopic gastrostomy prior to Glenn and hypothesize that those with a percutaneous endoscopic gastrostomy experience-enhanced interstage growth and reduced malnutrition rates.

Multisystem Inflammatory Syndrome in Children (MIS-C) Associated with 2019 Novel Coronavirus (SARS-CoV-2) Infection.

We report three critically ill pediatric patients (aged 6-10 years), presenting with features of multisystem inflammatory syndrome in children (MIS-C) from April 4 to May 10, 2020, to a tertiary-care center in New Jersey, United States. All patients tested positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection and were previously healthy. Clinical presentations were similar with fever, abdominal pain, gastrointestinal complaints, and/or rash.

Differentiating Flail Limb Syndrome From Amyotrophic Lateral Sclerosis.

Objective: The aim of the study was to investigate differences between flail limb syndrome and amyotrophic lateral sclerosis.

Design: A retrospective chart review identified 16 cases of amyotrophic lateral sclerosis and 16 of flail limb syndrome. Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, compound muscle action potential amsplitudes, and rate of loss of vital capacity were compared.

The Application of Next-generation Sequencing Tumor Molecular Profiling in the Diagnosis and Management of a Case of Acute Myelogenous Leukemia With MLL-PTD in a Pediatric Heart Transplant Recipient.

A 6-year-old girl with a history of heart transplantation was diagnosed with myelodysplastic syndrome, which progressed to acute myelogenous leukemia. Comprehensive genomic profiling of her tumor discovered an MLL-PTD (partial tandem duplication) and she received chemotherapy and a hematopoietic stem cell transplant (HSCT). She subsequently relapsed and tumor molecular profiling was repeated, revealing 2 new potentially targetable mutations (FLT3 and IDH2).

Clinical controversies in anticoagulation monitoring and antithrombin supplementation for ECMO.

During extracorporeal membrane oxygenation (ECMO), a delicate balance is required to titrate systemic anticoagulation to prevent thrombotic complications within the circuit and prevent bleeding in the patient. Despite focused efforts to achieve this balance, the frequency of both thrombotic and bleeding events remains high. Anticoagulation is complicated to manage in this population due to the complexities of the hemostatic system that are compounded by age-related developmental hemostatic changes, variable effects of the etiology of critical illness on hemostasis, and blood-circuit interaction.

Temporal trends in patient characteristics, injury mechanisms and outcomes in pediatric trauma admissions between 2010 and 2017.

Introduction: Injuries are a leading cause of preventable morbidity and mortality in children. Mechanisms of injuries and presentations are diverse in pediatric injuries and require special attention. Dedicated pediatric trauma care centers are ideal for management of children with injuries simultaneously serving as sources of research data.

Safety of dabigatran etexilate for the secondary prevention of venous thromboembolism in children.

This open-label, single-arm, prospective cohort trial is the first phase 3 safety study to describe outcomes in children treated with dabigatran etexilate for secondary venous thromboembolism (VTE) prevention. Eligible children aged 12 to <18 years (age stratum 1), 2 to <12 years (stratum 2), and >3 months to <2 years (stratum 3) had an objectively confirmed diagnosis of VTE treated with standard of care (SOC) for ≥3 months, or had completed dabigatran or SOC treatment in the DIVERSITY trial (NCT01895777) and had an unresolved clinical thrombosis risk factor requiring further anticoagulation. Children received dabigatran for up to 12 months, or less if the identified VTE clinical risk factor resolved.

Safety and tolerability of adjunctive lacosamide in a pediatric population with focal seizures - An open-label trial.

Purpose: To evaluate safety and tolerability of adjunctive lacosamide in children with focal seizures.

Methods: Patients were eligible for this open-label, fixed-titration trial (SP0847; NCT00938431) if aged 1 month-17 years with focal seizures taking 1-3 antiepileptic drugs. Findings from Cohort 1, aged 5-11 years, who received lacosamide ≤8 mg/kg/day, informed dosing for age-based cohorts 2-5, who then received ≤12 mg/kg/day (≤600 mg/day).

Financial Assistance for Fertility Preservation Among Adolescent and Young Adult Cancer Patients: A Utilization Review of the Sharing Hope/LIVESTRONG Fertility Financial Assistance Program.

Fertility preservation (FP) is a critical component of adolescent and young adult (AYA) cancer care that remains inadequately delivered. Prohibitive cost due to a lack of insurance coverage remains a barrier. Since 2004 Sharing Hope/LIVESTRONG Fertility has assisted AYA cancer patients through discounted FP rates and access to free medications.

Clinical Presentation and Outcome in Autoimmune Encephalitis Associated With N-Type Voltage-Gated Calcium Channels in Children.

Objective: We present the diagnostic and clinical course of the first multicenter case series of pediatric patients with autoimmune encephalitis associated with N-type voltage-gated calcium channel antibodies.

Methods: Data from 2 university hospitals were retrospectively reviewed and records of 3 patients with autoimmune encephalitis associated with N-type voltage-gated calcium channel antibodies were evaluated.

Results: The 3 pediatric patients (all female) had symptoms that spanned the clinical spectrum.

A Paradigm Shift in the Management of Cherubism? A Preliminary Report Using Imatinib.

Cherubism is an autosomal-dominant inherited mutation in the SH3BP2 gene on chromosome 4p16.3. It is characterized by bilateral symmetric fibro-osseous lesions that are limited to the maxilla and mandible.

Adjunctive Perampanel Oral Suspension in Pediatric Patients From ≥2 to <12 Years of Age With Epilepsy: Pharmacokinetics, Safety, Tolerability, and Efficacy.

Study 232, an open-label pilot study with an extension phase, evaluated the pharmacokinetics and preliminary safety/tolerability and efficacy of adjunctive perampanel oral suspension (≤0.18 mg/kg/d) in epilepsy patients aged ≥2 to <12 years. Patients were grouped into cohorts 1 (aged ≥7 to <12 years) and 2 (aged ≥2 to <7 years).

Pharmacological treatment of pediatric Gaucher disease.

Gaucher disease (GD) is an autosomal recessive disorder resulting from the deficiency of the lysosomal enzyme glucocerebrosidase (b-glucosidase), associated with varying degrees of visceral, bone and central nervous system pathology, leading to wide phenotypic diversity. Response to therapy and clinical outcomes are very different between the three clinical subtypes - non-neuronopathic, acute neuronopathic, and chronic neuronopathic forms; hence a definitive clinical diagnosis is essential. The availability of two therapeutic options, i.

Evaluation of long-term safety, tolerability, and behavioral outcomes with adjunctive rufinamide in pediatric patients (≥1 to <4 years old) with Lennox-Gastaut syndrome: Final results from randomized study 303.

Objective: Evaluate the long-term safety, tolerability, and behavioral effects of adjunctive rufinamide in pediatric patients (≥1 to <4 years old) with inadequately controlled seizures associated with Lennox-Gastaut syndrome (LGS).

Methods: Study 303 (ClinicalTrials.gov identifier NCT01405053) was a multicenter, randomized, open-label, Phase III trial.

Diagnosis and Management of Mild Traumatic Brain Injury in Children: A Systematic Review.

Importance: In recent years, there has been an exponential increase in the research guiding pediatric mild traumatic brain injury (mTBI) clinical management, in large part because of heightened concerns about the consequences of mTBI, also known as concussion, in children. The CDC National Center for Injury Prevention and Control's (NCIPC) Board of Scientific Counselors (BSC), a federal advisory committee, established the Pediatric Mild TBI Guideline workgroup to complete this systematic review summarizing the first 25 years of literature in this field of study.

Objective: To conduct a systematic review of the pediatric mTBI literature to serve as the foundation for an evidence-based guideline with clinical recommendations associated with the diagnosis and management of pediatric mTBI.

Centers for Disease Control and Prevention Guideline on the Diagnosis and Management of Mild Traumatic Brain Injury Among Children.

Importance: Mild traumatic brain injury (mTBI), or concussion, in children is a rapidly growing public health concern because epidemiologic data indicate a marked increase in the number of emergency department visits for mTBI over the past decade. However, no evidence-based clinical guidelines have been developed to date for diagnosing and managing pediatric mTBI in the United States.

Objective: To provide a guideline based on a previous systematic review of the literature to obtain and assess evidence toward developing clinical recommendations for health care professionals related to the diagnosis, prognosis, and management/treatment of pediatric mTBI.

Expanding the clinical phenotype of individuals with a 3-bp in-frame deletion of the NF1 gene (c.2970_2972del): an update of genotype-phenotype correlation.

Purpose: Neurofibromatosis type 1 (NF1) is characterized by a highly variable clinical presentation, but almost all NF1-affected adults present with cutaneous and/or subcutaneous neurofibromas. Exceptions are individuals heterozygous for the NF1 in-frame deletion, c.2970_2972del (p.

Pediatric Methicillin-Resistant Osteoarticular Infections.

Osteoarticular infections (OSI) are a significant cause of hospitalizations and morbidity in young children. The pediatric patient with OSI presents unique challenges in diagnosis and management due to higher morbidity, effect on growth plate with associated long-lasting sequelae, and challenges in early identification and management. Methicillin-resistant (MRSA), first described in the 1960s, has evolved rapidly to emerge as a predominant cause of OSI in children, and therefore empiric treatment for OSI should include an antibiotic effective against MRSA.

Spotlight on taliglucerase alfa in the treatment of pediatric patients with type 1 Gaucher disease.

Gaucher disease (GD) is a heritable storage disorder caused by functional defects of the lysosomal acid β-glucosidase and the accumulation of glucosylceramide within macrophages, resulting in multiple organ dysfunction. There are three commercially available enzyme replacement therapy (ERT) products for the treatment of GD type 1 (GD1): imiglucerase, velaglucerase alfa, and taliglucerase alfa. Imiglucerase and velaglucerase alfa are produced in different mammalian cell systems; imiglucerase requires postproduction deglycosylation to expose terminal α-mannose residues, which are required for mannose receptor-mediated uptake by target macrophages.