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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.
Sports practice should be supported in people with severe hemophilia, but patient counseling and tailoring prophylactic treatment with clotting factors and non-replacement therapy is essential.
Expert Rev Hematol
January 2025
Department of Physical and Rehabilitation Medicine, Hospital Universitario La Paz, Madrid, Spain.
Introduction: This article discusses the current role of sports practice in people with hemophilia (PWH).
Areas Covered: On 11 January 2025, a bibliographic search was carried out in PubMed using 'hemophilia sports' as keywords. A total of 411 articles were found, of which only 22 were finally analyzed because they were directly related to the title of this article (inclusion criterion).
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 PDFA Case Report of Cryoneurolysis With Factor VIII Administration for Cerebral Palsy-related Spasticity in a Patient With Hemophilia A.
Adv Rehabil Sci Pract
January 2025
Vancouver Island Health Authority, Victoria, BC, Canada.
Spasticity affects up to 80% of individuals with cerebral palsy and can lead to pain and difficulties with performing activities of daily living. If left untreated, spasticity can progress to contracture and neuro-orthopedic deformities. Cryoneurolysis is an emerging and mini-invasive ultrasound-guided technique that causes secondary axonotmesis of peripheral nerves through the formation of an ice ball and may result in months to years of improved range of motion and reduced pain in patients with spasticity.
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