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Phase 1 Study of Quercetin, a Natural Antioxidant in Children and Young Adults with Fanconi Anemia (FA).
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Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States.
Fanconi anemia (FA) is a rare inherited disorder characterized by progressive bone marrow failure (BMF) and a predisposition to malignancy. Systemic reactive-oxygen species (ROS) and increased sensitivity of FA hematopoietic progenitors to ROS play a key role in the pathogenesis of BMF. Treatment with antioxidants improve hematopoietic function in Fancc-/- mice.
View Article and Find Full Text PDFSafety and efficacy of trifluridine/tipiracil +/- bevacizumab plus XB2001 (anti-IL-1α antibody): a single-center phase 1 trial.
Signal Transduct Target Ther
January 2025
Centre de Recherche INSERM Center for Translational and Molecular Medicine, 21000, Dijon, France.
In the tumour microenvironment, IL-1α promotes neoangiogenesis, matrix remodelling, tumour proliferation, chemoresistance, and metastases. Highly expressed in human colorectal cancers, IL-1α is associated with poor prognosis. XB2001, a fully human monoclonal antibody neutralizing IL-1α, was evaluated for safety and preliminary efficacy with trifluridine/tipiracil (FTD/TPI) and bevacizumab in metastatic colorectal cancer patients previously treated with oxaliplatin- and irinotecan-based chemotherapies.
View Article and Find Full Text PDFThe clinical presentation and genetic diagnosis of Tangier disease in the pediatric age group.
J Pediatr Endocrinol Metab
January 2025
Division of Pediatric Neurology, Department of Pediatrics and Child Health, Erciyes University, Faculty of Medicine, Kayseri, Türkiye.
Objectives: Tangier disease (TD) is a rare autosomal recessive condition characterized by high-density lipoprotein (HDL) deficiency; involving symptoms of polyneuropathy, hyperplastic orange-yellow tonsils, vision disorder, and sudden cardiac death. The major clinical symptoms of TD may not all be co-present. This study evaluates patients diagnosed with TD in childhood to improve the possibility of early diagnosis of asymptomatic cases by reporting our patients' clinical characteristics in order to minimize delayed diagnosis and emphasize the importance of TD, easily detected by HDL measurement.
View Article and Find Full Text PDFCommon intestinal parasitic infections in an improved water access, sanitation, and hygiene profile setting in North Jakarta, Indonesia.
Narra J
December 2024
Master of Biomedicine Study Program, School of Medicine and Health Sciences, Universitas Katolik Indonesia Atma Jaya, Jakarta, Indonesia.
Intestinal parasitic infections (IPIs), caused by helminths and/or protozoa, continue to be a significant public health concern in Indonesia. Water access, sanitation, and hygiene practices (WASH) are influential factors for IPIs, especially among children. The aim of this study was to investigate the association between WASH and IPIs among school-aged children.
View Article and Find Full Text PDFHorse antithymocyte globulin test doses and infusion reactions in adults with aplastic anemia: A multicenter retrospective experience.
J Oncol Pharm Pract
January 2025
Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, Ann Arbor, MI, USA.
Introduction: Horse antithymocyte globulin carries a black box warning for life-threatening anaphylactic reactions, and prescribing information recommends test doses to identify patients at highest risk of this adverse effect. The predictive value of such test doses is not well validated, and practicality of use is unclear.
Methods: This was a planned secondary analysis of a multicenter, retrospective cohort study in adults with severe aplastic anemia being managed with horse antithymocyte globulin as part of their treatment regimen.
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