Factor VIII or IX replacement in a prophylactic manner is utilized for many patients with moderate to severe hemophilia A or B. Studies have shown it to be effective in reducing or preventing degenerative joint disease in many but not all patients. However, many unanswered questions still exist and optimization of this expensive treatment regimen is needed. This paper recounts the current products that are available for use and explores the literature regarding different treatment regimens. It explores age at initiation, dose, interval between infusions, joint health outcomes, barriers to compliance and age at discontinuation of prophylaxis. Individualized treatment is recommended. Collaborative efforts are needed to improve outcomes for all persons with hemophilia.
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http://dx.doi.org/10.1159/000090930 | DOI Listing |
Acta Orthop Traumatol Turc
December 2024
Department of Orthopedics and Traumatology, Ege University School of Medicine, Izmir, Türkiye.
Objective: This study aimed to compare preoperative and postoperative measures in haemophiliacs who had simultaneous total hip and knee arthroplasties.
Methods: A retrospective database search identified five patients with severe factor 8 deficiencies who underwent simultaneous hip and knee joint replacement surgery between 2002-2018. Preoperative and postoperative evaluations included Harris Hip Score (HHS), Knee Society Score (KSS), Knee Injury and Osteoarthritis Outcome Score (KOOS), range of motion, flexion contracture (FC), Visual Analog Scale (VAS), hip-knee angle, and leg length discrepancy.
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.
Cells
January 2025
Research Department, Sidra Medicine, Doha P.O. Box 26999, Qatar.
Hemophilia A (HA) is associated with FVIII coagulation insufficiency or inactivity leading to excessive bleeding. Elevated FVIII, on the contrary, is associated with thrombophilia, thrombosis, myocardial infarctions, and stroke. Active FVIII (aFVIII) uses its C2 domain to bind to blood cells' membranes, consequently carrying out its coagulative function.
View Article and Find Full Text PDFRes 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 PDFRegen Ther
March 2025
Pediatric Cell and Gene Therapy Research Center, Gene, Cell & Tissue Research Institute, Tehran University of Medical Science, Tehran, Iran.
Gene therapy (GT) as a groundbreaking approach holds promise for treating many diseases including immune deficiencies and blood disorders. GT can benefit patients suffering from these diseases, especially those without matched donors or who are at risk after hematopoietic stem cell transplantation (HSCT). Due to all the advances in the field of GT, its main challenge is still gene delivery.
View Article and Find Full Text PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!