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Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France.
Ther Adv Hematol
January 2025
Sobi, Basel, Switzerland.
Background: More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.
Objectives: We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.
Methods: Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE.
A 5-year Single-center Experience on the Use of Emicizumab Prophylaxis in Children With Severe Hemophilia A With and Without Factor VIII Inhibitors.
J Pediatr Hematol Oncol
January 2025
Department of Paediatric Haematology, NHS Foundation Trust, Birmingham Children's Hospital, Birmingham, UK.
Objective: Emicizumab promotes efficacious hemostasis in persons with hemophilia A persons with hemophilia A with and without inhibitors. Primary analyses of real-world data and clinical trials have shown emicizumab efficacy and safety; however, long-term data are limited.
Methods: This retrospective study was conducted to assess real-world long-term outcomes of pediatric patients on emicizumab in our hemophilia center between the period of February 2018 and September 2023.
No correlation between thrombin generation and emicizumab levels: implications for monitoring emicizumab therapy.
Res Pract Thromb Haemost
January 2025
Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
Background: Emicizumab, a bispecific antibody that mimics factor (F)VIII, has significantly improved hemophilia A management. Although emicizumab levels can be measured, tools for estimating the hemostatic efficacy of emicizumab are lacking. Thrombin generation (TG) assays can distinguish bleeding phenotypes in persons with hemophilia A on FVIII prophylaxis and may also be used during emicizumab therapy.
View Article and Find Full Text PDFAdipose derived stromal vascular fraction cells therapy in hemophilic arthropathy: A case report.
Agri
January 2025
Department of Anesthesiology and Reanimation, Ege University Faculty of Medicine, İzmir, Türkiye.
Stromal vascular fraction (SVF) is a heterogeneous collection of cells obtained from adipose tissue through lipoaspiration and is an alter-native intraarticular treatment option, especially in osteoarthritis (OA). The anti-inflammatory and extracellular tissue repair-stimulating properties of SVF increase its effectiveness in regeneration and repair mechanisms. One of the most common symptoms of hemophilia A and B is hemophilic arthropathy (HA).
View Article and Find Full Text PDFAn investigation of a hemophilia A female with heterozygous intron 22 inversion and skewed X chromosome inactivation.
Front Genet
January 2025
Department of Hematology, The Second Affiliated Hospital, Chongqing Medical University, Chongqing, China.
Objectives: Hemophilia A (HA) is an X-linked recessive inherited bleeding disorder that typically affects men. Women are usually asymptomatic carriers, and rarely presenting with severe or moderately severe phenotype. This study aims to describe a case of a 17-year-old girl with moderate HA, investigating the mechanisms of her condition and the genetic basis within her family.
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