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BRAF inhibitor monotherapy in BRAFV600E-mutated pediatric low-grade glioma: a single center's experience.
Front Oncol
January 2025
Department of Pediatrics, Children's Healthcare of Atlanta, Atlanta, GA, United States.
Background: Pediatric low-grade gliomas (pLGGs) have an overall survival of over 90%; however, patients harboring a BRAF alteration may have worse outcomes, particularly when treated with classic chemotherapy. Combined BRAF/MEK inhibition following incomplete resection demonstrated improved outcome in BRAF altered pLGG compared to combined carboplatin/vincristine chemotherapy and is now considered the standard FDA-approved treatment for this group of tumors. The aim herein was to investigate the efficacy and tolerability of single agent BRAF inhibitor treatment in BRAF altered pLGG.
View Article and Find Full Text PDFAssociation between extracellular DNA levels, markers of inflammation and left ventricular mass index in children with chronic kidney disease.
Sci Rep
January 2025
Division of Pediatrics, Department of Clinical Science, Intervention and Technology, Karolinska Institutet, Stockholm, Sweden.
Chronic kidney disease (CKD) is associated with chronic low-grade inflammation, but the primary factors triggering this inflammation remain unclear. Extracellular or cell-free DNA (exDNA) originates from virtually all tissues, being released during cell death, and stimulates the innate immune system. Our study was designed as an observational, cross-sectional cohort study of children with CKD (both before and after kidney transplantation) and controls to analyze associations between exDNA, markers of inflammation, and cardiovascular health.
View Article and Find Full Text PDFPoor muscle quality: A hidden and detrimental health condition in obesity.
Rev Endocr Metab Disord
January 2025
Human Nutrition Research Unit, Department of Agricultural, Food and Nutritional Sciences, University of Alberta, Edmonton, AB, Canada.
Poor muscle quality (MQ) is a hidden health condition in obesity, commonly disregarded and underdiagnosed, associated with poor health-related outcomes. This narrative review provides an in-depth exploration of MQ in obesity, including definitions, available assessment methods and challenges, pathophysiology, association with health outcomes, and potential interventions. MQ is a broad term that can include imaging, histological, functional, or metabolic assessments, evaluating beyond muscle quantity.
View Article and Find Full Text PDFFDA Approval Summary: Tovorafenib for Relapsed or Refractory BRAF-altered Pediatric Low-Grade Glioma.
Clin Cancer Res
January 2025
United States Food and Drug Administration, Silver Spring, Maryland, United States.
On April 23, 2024, FDA granted accelerated approval to tovorafenib, a type II RAF kinase inhibitor, for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. Efficacy was evaluated in FIREFLY-1 (NCT04775485), a single-arm, open-label, multicenter trial that enrolled patients 6 months to 25 years of age with relapsed or refractory pLGG with an activating BRAF alteration who had received prior systemic therapy. The major efficacy outcome measure was radiologic overall response rate (ORR), defined as the proportion of patients with complete response, partial response, or minor response as determined by blinded independent central review using Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria.
View Article and Find Full Text PDFAn Acta Orthopaedica educational article: Treatment of pediatric spondylolysis and spondylolisthesis.
Acta Orthop
January 2025
Helsinki New Children's Hospital, Helsinki University Hospital, Helsinki, Finland.
Spondylolysis is defined as a defect or elongation in the pars interarticularis of the lumbar spine, either unilateral or bilateral. Growing children with bilateral spondylolysis may develop spondylolisthesis, i.e.
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