148 results match your criteria: "Center for Neuroscience and Behavioral Medicine[Affiliation]"
Background: Congenital heart disease (CHD) is a risk factor for developmental delay and for attention-deficit hyperactivity disorder (ADHD). The Cardiac Neurodevelopmental Outcome Collaborative has developed recommendations for ongoing monitoring of this at-risk population to be able to detect developmental, learning, and behavioral concerns, as they become apparent as a child ages.
Case Presentation: A 4-year-old boy with tetralogy of Fallot with a ventricular septal defect repaired in infancy was followed periodically in the cardiac neurodevelopmental follow-up clinic and diagnosed with autism spectrum disorder as well as additional developmental and medical issues.
Neuro Oncol
October 2024
Department of Pediatric Oncology, Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts, USA.
Adolescents and young adults (AYAs; ages 15-39 years) are a vulnerable population facing challenges in oncological care, including access to specialized care, transition of care, unique tumor biology, and poor representation in clinical trials. Brain tumors are the second most common tumor type in AYA, with malignant brain tumors being the most common cause of cancer-related death. The 2021 WHO Classification for central nervous system (CNS) Tumors highlights the importance of integrated molecular characterization with histologic diagnosis in several tumors relevant to the AYA population.
View Article and Find Full Text PDFPediatr Blood Cancer
November 2024
Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA.
Objectives: Survivors of childhood B-acute lymphoblastic leukemia (B-ALL) are at risk for difficulties with attention and executive functioning (EF) as a late effect of treatment. The present study aimed to identify treatment and demographic factors associated with risk for difficulties with EF in youth treated for high-risk B-ALL.
Method: Children and adolescents with B-ALL treated on Children's Oncology Group (COG) protocol AALL0232 were randomized to high-dose or escalating-dose methotrexate (MTX), and either dexamethasone or prednisone during the induction phase.
Neurocrit Care
August 2024
Division of Neurogenetics and Neurodevelopmental Pediatrics, Center for Neuroscience and Behavioral Medicine, GWU School of Medicine and Health Sciences, Children's National Hospital, 111 Michigan Ave, NW, Washington, DC, 20010, USA.
Background: Acute metabolic crises in inborn errors of metabolism (such as urea cycle disorders, organic acidemia, maple syrup urine disease, and mitochondrial disorders) are neurological emergencies requiring management in the pediatric intensive care unit (PICU). There is a paucity of data pertaining to electroencephalograms (EEG) characteristics in this cohort. We hypothesized that the incidence of background abnormalities and seizures in this cohort would be high.
View Article and Find Full Text PDFPediatr Neurol
October 2024
Division of Neurogenetics & Developmental Pediatrics, Center for Neuroscience and Behavioral Medicine, Children's National Medical Center, Washington, District of Columbia. Electronic address:
Background: This retrospective clinical study performed at a single clinical center aimed to identify the prevalence of seizures in individuals with urea cycle disorders (UCDs) with and without hyperammonemic (HA) crises. In addition, we sought to correlate the utility of biochemical markers and electroencephalography (EEG) in detecting subclinical seizures during HA.
Methods: Medical records of individuals with UCDs enrolled in Urea Cycle Disorders Consortium Longitudinal Study (UCDC-LS) (NCT00237315) at Children's National Hospital between 2006 and 2022 were reviewed for evidence of clinical and subclinical seizuress during HA crises, and initial biochemical levels concurrently.
Int J Comput Assist Radiol Surg
October 2024
Division of Neurosurgery, Center for Neuroscience and Behavioral Medicine, Children's National Hospital, Washington, DC, USA.
Purpose: Lumbar discectomy is among the most common spine procedures in the US, with 300,000 procedures performed each year. Like other surgical procedures, this procedure is not excluded from potential complications. This paper presents a video annotation methodology for microdiscectomy including the development of a surgical workflow.
View Article and Find Full Text PDFJCO Precis Oncol
June 2024
Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.
Purpose: Midline low-grade gliomas (mLGGs) of early childhood have a poorer prognosis compared with tumors of other localizations and in older patients. LGGs are associated with aberrant activation of RAS-RAF-MEK pathway, and pharmacological inhibition of the pathway has therapeutic promise. The aim of this study was clinical and molecular characterization of infantile mLGGs, with emphasis on the efficacy of targeted kinase inhibition.
View Article and Find Full Text PDFAm J Med Genet A
November 2024
Neurogenetics Clinic Co-Director, Center for Neuroscience and Behavioral Medicine, Washington, DC, USA.
Pseudo-TORCH Syndrome (PTS) encompasses a heterogeneous group of genetic disorders that may clinically and radiologically resemble congenital TORCH infections. These mimickers present with overlapping features manifested as intracranial and systemic abnormalities. Collagen type IV alpha 1 chain (COL4A1)-related diseases, characterized by autosomal dominant inheritance, exhibit a diverse phenotypic spectrum involving cerebrovascular, renal, ophthalmological, cardiac, and muscular abnormalities.
View Article and Find Full Text PDFPediatr Blood Cancer
September 2024
Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.
Vision (Basel)
May 2024
Retinal Neurophysiology Section, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Neurofibromatosis type 1 (NF1) is an inherited autosomal dominant disorder primarily affecting children and adolescents characterized by multisystemic clinical manifestations. Mutations in neurofibromin, the protein encoded by the tumor suppressor gene, result in dysregulation of the RAS/MAPK pathway leading to uncontrolled cell growth and migration. Neurofibromin is highly expressed in several cell lineages including melanocytes, glial cells, neurons, and Schwann cells.
View Article and Find Full Text PDFJ Child Neurol
May 2024
Center for Neuroscience and Behavioral Medicine, Children's National Hospital, Washington, DC, USA.
Children with developmental disabilities have increased risk of epilepsy and need for overnight video electroencephalographic (EEG) monitoring. However, video EEGs have historically been considered difficult to complete for this population. An autism support service at a pediatric tertiary care hospital implemented a coordinated team approach to help children with developmental disability tolerate overnight video EEGs.
View Article and Find Full Text PDFNeuro Oncol
August 2024
UCL Great Ormond Street Institute of Child Health, Great Ormond Street Hospital for Children, London, UK.
Pediatric low-grade glioma (pLGG) is the most common childhood brain tumor group. The natural history, when curative resection is not possible, is one of a chronic disease with periods of tumor stability and episodes of tumor progression. While there is a high overall survival rate, many patients experience significant and potentially lifelong morbidities.
View Article and Find Full Text PDFRes Pract Thromb Haemost
February 2024
Cortica Healthcare and Children's Hospital Los Angeles, Westlake Village, California, USA.
Background: Nonacog beta pegol (N9-GP) is an extended half-life PEGylated factor (F)IX product with established efficacy and short-term safety in persons with hemophilia B (HB). Long-term safety has been evaluated for polyethylene glycol exposure but not N9-GP.
Objectives: To assess safety, neurodevelopmental, and efficacy outcomes of children with HB receiving N9-GP prophylaxis across 2 open-label, single-arm, phase 3 studies: paradigm5 (previously treated patients [PTPs]) and paradigm6 (previously untreated patients [PUPs]) in this interim analysis.
Nat Med
May 2024
Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
Clin Cancer Res
June 2024
Center for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.
In October 2022, the FDA Oncology Center of Excellence hosted an educational symposium entitled, "Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma (pLGG) Clinical Trials." The symposium brought together patient advocates, regulators from the FDA and the European Medicines Agency (EMA), and an international group of academic thought leaders in the field of pediatric neuro-oncology to discuss the potential role of functional outcomes, including visual acuity, motor function, and neurocognitive performance, as endpoints in clinical trials enrolling patients with pLGG. The panel discussed challenges and opportunities regarding the selection, implementation, and evaluation of clinical outcome assessments in these functional domains and outlined key considerations for their inclusion in future clinical trial design and role in new drug development.
View Article and Find Full Text PDFNeuro Oncol
March 2024
Hopp Children's Cancer Center (KiTZ), National Center for Tumor Diseases (NCT), German Cancer Research Center (DKFZ) and University Hospital, Heidelberg, Germany.
Within the last few decades, we have witnessed tremendous advancements in the study of pediatric low-grade gliomas (pLGG), leading to a much-improved understanding of their molecular underpinnings. Consequently, we have achieved successful milestones in developing and implementing targeted therapeutic agents for treating these tumors. However, the community continues to face many unknowns when it comes to the most effective clinical implementation of these novel targeted inhibitors or combinations thereof.
View Article and Find Full Text PDFNat Med
January 2024
Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
Curr Neurol Neurosci Rep
December 2023
Brain Tumor Institute, Children's National Hospital, Washington D C, USA.
Purpose Of Review: Review recent advances in the understanding of pediatric medulloblastoma including etiology, biology, radiology, and management of pediatric medulloblastoma.
Recent Findings: The classic four subgroups have been reclassified and further subdivided based on new molecular findings. Research is revealing the cell origins of the different subtypes of medulloblastoma.
Genet Med Open
September 2023
Division of Neurogenetics and Neurodevelopmental Pediatrics, Center for Neuroscience and Behavioral Medicine, Children's National Hospital, Washington, DC.
Cancers (Basel)
July 2023
Murdoch Children's Research Institute, Parkville, VIC 3052, Australia.
Despite the evidence of elevated autistic behaviors and co-occurring neurodevelopmental difficulties in many children with neurofibromatosis type 1 (NF1), we have a limited understanding of the sensory processing challenges that may occur with the condition. This study examined the sensory profile of children and adolescents with NF1 and investigated the relationships between the sensory profiles and patient characteristics and neuropsychological functioning. The parent/caregivers of 152 children with NF1 and 96 typically developing children completed the Sensory Profile 2 (SP2), along with standardized questionnaires assessing autistic behaviors, ADHD symptoms, internalizing symptoms, adaptive functioning, and social skills.
View Article and Find Full Text PDFInt J Comput Assist Radiol Surg
September 2023
Georgetown University School of Medicine, Washington, DC, USA.
Purpose: Surgical data science is an emerging field focused on quantitative analysis of pre-, intra-, and postoperative patient data (Maier-Hein et al. in Med Image Anal 76: 102306, 2022). Data science approaches can decompose complex procedures, train surgical novices, assess outcomes of actions, and create predictive models of surgical outcomes (Marcus et al.
View Article and Find Full Text PDFJ Pathol
July 2023
Center for Genetics Medicine Research, Children's National Hospital, Washington, DC, USA.
The molecular characteristics of pediatric brain tumors have not only allowed for tumor subgrouping but have led to the introduction of novel treatment options for patients with specific tumor alterations. Therefore, an accurate histologic and molecular diagnosis is critical for optimized management of all pediatric patients with brain tumors, including central nervous system embryonal tumors. We present a case where optical genome mapping identified a ZNF532::NUTM1 fusion in a patient with a unique tumor best characterized histologically as a central nervous system embryonal tumor with rhabdoid features.
View Article and Find Full Text PDFSeizure
March 2023
Department of Health Studies, American University, Washington DC, United States; Department of Neuroscience, American University, Washington DC, United States; Center for Neuroscience and Behavior, American University, Washington DC, United States. Electronic address:
Neuro Oncol
February 2023
Division of Hematology-Oncology, Department of Pediatrics, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.
Background: Craniopharyngioma is a histologically benign tumor of the suprasellar region for which survival is excellent but quality of life is often poor secondary to functional deficits from tumor and treatment. Standard therapy consists of maximal safe resection with or without radiation therapy. Few prospective trials have been performed, and response assessment has not been standardized.
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