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Hemophilia A is a clotting disorder due to factor VIII deficiency, leading to prolonged bleeding. Acquired hemophilia A results from the immune system attacking factor VIII, typically occurring later in life. Factor V Leiden is a genetic mutation causing abnormal blood clot formation, primarily in veins.
View Article and Find Full Text PDFSafety and efficacy of valoctocogene roxaparvovec with prophylactic glucocorticoids: 1-year results from the phase 3b, single-arm, open-label GENEr8-3 study.
J Thromb Haemost
January 2025
BioMarin Pharmaceutical Inc., Novato, CA, USA.
Background: Valoctocogene roxaparvovec, an adeno-associated virus vector that transfers a human factor VIII (FVIII) coding sequence to hepatocytes, provides bleeding protection for people with severe hemophilia A (HA).
Objective: Determine the efficacy and safety of valoctocogene roxaparvovec with concomitant prophylactic glucocorticoids in the open-label, single-arm, phase 3b GENEr8-3 trial.
Methods: Participants with severe HA who were using HA prophylaxis received one 6x10 vg/kg infusion of valoctocogene roxaparvovec concomitantly with daily prophylactic glucocorticoids (40 mg prednisolone equivalent/d weeks 0‒8; taper to 5 mg/d weeks 9‒19).
Hematologic Complications of Pregnancy.
Eur J Haematol
January 2025
Division of Hematology and Medical Oncology, Oregon Health & Science University, Portland, Oregon, USA.
Hematologic complications are common in pregnancy and can significantly impact both maternal and fetal health. Recognizing and treating these complications can be challenging due to the limited evidence available to guide clinical consultants. Iron deficiency anemia is the most prevalent hematologic issue in pregnancy and often occurs due to increased maternal blood volume and the nutritional demands of the growing fetus.
View Article and Find Full Text PDFGenotyping as Part of Routine Clinical Care-The Outcomes for a Large Paediatric Vascular Anomaly Cohort.
Am J Med Genet A
January 2025
Genetic Health Queensland, Royal Brisbane and Women's Hospital, Herston, Australia.
We describe the phenotypic and genotypic spectrum of patients with vascular anomaly (VA) in a paediatric multi-disciplinary VA clinic. We measured the clinical utility of genotyping by comparing pre and posttest diagnosis and management. A 46-month retrospective analysis occurred for 250 patients offered genetic testing in the VA clinic.
View Article and Find Full Text PDF[Clinical Analysis of 25 Cases of Acquired Hemophilia A in a Single Center].
Zhongguo Shi Yan Xue Ye Xue Za Zhi
December 2024
Department of Hematology, The Second Hospital of Hebei Medical University, Hebei Key Laboratory of Hematology, Shijiazhuang 050000, Hebei Province, China.
Objective: To explore the diagnosis and treatment of acquired hemophilia A (AHA) based on the analysis of clinical data.
Methods: A retrospective analysis was conducted on the clinical manifestations, laboratory characteristics, treatment, and outcomes of 25 patients diagnosed with AHA who were admitted to the Second Hospital of Hebei Medical University.
Results: Among all patients, 11 cases had secondary factors, including 5 cases of autoimmune diseases, 3 cases of pregnancy-related disease, 1 case of pemphigoid, 1 case of Graves' disease, and 1 case of monoclonal gammaglobulinemia of unknown significance (MGUS).
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