Aggressive histiocytic lesions are uncommon in the pediatric population. These neoplasms occur in isolation or after therapy for other types of hematopoietic malignancy such as T-cell acute lymphoblastic leukemia. The etiology of these lesions is poorly understood, and no definitive standard of care has been established for patients with these diagnoses. Here, we report the success of thalidomide treatment for 2 subtypes of histiocytic proliferation--metastatic histiocytic sarcoma and extracutaneous juvenile xanthogranuloma--in pediatric patients. Our findings highlight the importance of considering thalidomide therapy in this unique and difficult to treat patient population.
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http://dx.doi.org/10.1097/MPH.0b013e3182580d6e | DOI Listing |
World J Gastroenterol
January 2025
Department of Gastroenterology, Air Force Medical Center, Beijing 100142, China.
Background: Gastrointestinal (GI) vascular bleeding disorders pose significant clinical challenges due to their complex pathogenesis and varied treatment responses. Despite advancements in diagnostic and therapeutic techniques, optimal management strategies remain elusive, necessitating further research.
Aim: To assess research trends and clinical advancements in GI vascular bleeding disorders, highlighting key themes and therapeutic progress.
Sci Rep
January 2025
Jiangsu Key Laboratory of Marine Pharmaceutical Compound Screening, College of Pharmacy, Jiangsu Ocean University, Lianyungang, 222005, China.
The ubiquitin-proteasome system (UPS) is essential for cellular homeostasis, regulating the degradation of proteins involved in key processes such as cell cycle, apoptosis, and DNA repair. Dysregulation of the UPS is implicated in hepatocellular carcinoma (HCC), contributing to tumor progression and therapeutic resistance. The cereblon (CRBN) E3 ubiquitin ligase complex is a crucial component of the UPS, particularly in modulating protein degradation in response to small-molecule modulators like thalidomide.
View Article and Find Full Text PDFCureus
December 2024
Department of Pediatrics, The Jikei University School of Medicine, Tokyo, JPN.
Congenital intracranial hemangiomas are rare benign vascular tumors that develop before birth. Although various treatments, including surgery, steroids, interferon-α, thalidomide, bevacizumab, or propranolol, have been reported, no standard therapy has been established. We report the case of a neonate with congenital intracranial hemangioma and central nervous system symptoms requiring therapeutic intervention.
View Article and Find Full Text PDFJ Med Chem
January 2025
Department of Chemical Biology and Therapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, United States.
PROTACs usually occupy physicochemical space outside the one defined by classical drug-like molecules, which often presents considerable challenges in their optimization and development for oral administration. We have previously reported phenyl glutarimide (PG)-based BET PROTAC SJ995973, with improved overall degradation and antiproliferative activities compared to its direct thalidomide-based analogue dBET1, but similarly poor pharmacokinetic profile. To further demonstrate the PG utility, we describe here optimization efforts that led to the discovery of an orally bioavailable BET-PROTAC SJ44236 (), and results of a comprehensive comparative study with analogues containing alternative CRBN-directing warheads.
View Article and Find Full Text PDFBackground: Psoriasis is a chronic, systemic, inflammatory skin disease, with increasing prevalence; however, few studies have reported real-world prescription patterns and healthcare burden.
Objectives: This retrospective, observational cohort study used statutory health insurance claims data (January 2014-December 2019) to estimate prevalence/incidence of moderate-to-severe psoriasis in Germany. Patient characteristics, treatment patterns/compliance, and healthcare resource utilization (HCRU)/costs were evaluated, focusing on apremilast and anti-interleukin (IL), and anti-tumor necrosis factor (TNF) biologics.
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