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Effectiveness of potent topical corticosteroids versus mild ones in primary care for children with moderate flare-ups of atopic dermatitis; results of a randomised controlled trial.
BMJ Open
December 2024
Department Dermatology, Center of Pediatric Dermatology, Erasmus MC University Medical Center-Sophia Children's Hospital, Rotterdam, the Netherlands.
Objective: To assess the effectiveness of a potent topical corticosteroid (TCS) as an initial treatment in primary care for children with moderate flare-ups of atopic dermatitis (AD), compared to starting on a mild TCS.
Design: An observational prospective cohort study with an embedded pragmatic multicentre open-label randomised controlled trial.
Setting: A total of 53 general practices in the southwest of the Netherlands took part in the study.
Randomised, placebo-controlled, double-blinded, four-way crossover trial to demonstrate the comparative pharmacodynamic equivalence of a non-invasive diagnostic test for adrenal insufficiency in a healthy population: the STARLIT-2 study protocol.
BMJ Open
December 2024
Clinical Medicine, School of Medicine and Population Health, The University of Sheffield, Sheffield, UK
Introduction: Cortisol is an essential stress hormone and failure of its production, known as adrenal insufficiency (AI), is associated with significant mortality due to adrenal crisis. The Short Synacthen Test (SST) is the current diagnostic test of choice for AI, but it is both invasive and resource intensive. Globally, there is an unmet need for a non-invasive, cost-effective test.
View Article and Find Full Text PDFImpact of sex hormones on pheochromocytomas, paragangliomas, and gastroenteropancreatic neuroendocrine tumors.
Eur J Endocrinol
January 2025
Department of Internal Medicine IV, LMU University Hospital, LMU Munich, 80336 Munich, Germany.
Objective: The effects of sex hormones remain largely unexplored in pheochromocytomas and paragangliomas (PPGLs) and gastroenteropancreatic neuroendocrine tumors (GEP-NETs).
Methods: We evaluated the effects of estradiol, progesterone, Dehydroepiandrosterone sulfate (DHEAS), and testosterone on human patient-derived PPGL/GEP-NET primary culture cell viability (n = 38/n = 12), performed next-generation sequencing and immunohistochemical hormone receptor analysis in patient-derived PPGL tumor tissues (n = 36).
Results: In PPGLs, estradiol and progesterone (1 µm) demonstrated overall significant antitumor effects with the strongest efficacy in PPGLs with NF1 (cluster 2) pathogenic variants.
Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study.
Neurology
January 2025
The Dubowitz Neuromuscular Centre, Developmental Neurosciences Department, University College London, Great Ormond Street Institute of Child Health, United Kingdom.
Background And Objectives: Safety and efficacy of IV onasemnogene abeparvovec has been demonstrated for patients with spinal muscular atrophy (SMA) weighing <8.5 kg. SMART was the first clinical trial to evaluate onasemnogene abeparvovec for participants weighing 8.
View Article and Find Full Text PDFMetastatic Neuroblastoma Masquerading as Ewing's Sarcoma on X-ray: An Imaging Puzzle in Pediatric Oncology.
J Orthop Case Rep
January 2025
Department of Pathology, All India Institute of Medical Sciences Bhopal, Bhopal, Madhya Pradesh, India.
Introduction: Neuroblastoma is an embryonic tumor of the peripheral sympathetic nervous system. It is the most common extracranial solid tumor of childhood and accounts for up to 15% of all pediatric cancer fatalities. The manifestation of neuroblastoma is variable depending on the location of the tumor and the presence or absence of paraneoplastic syndromes.
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