Valoctocogene Roxaparvovec May Provide Lower Bleeding Rates Than Emicizumab in Severe Hemophilia A
In patients with severe hemophilia A, prophylactic treatment with valoctocogene roxaparvovec gene therapy provided a lower annualized bleeding rate ...
Switching to Different FVIII Product Not Associated with Anti-FVIII Inhibitor Development
Individuals with hemophilia A who switched to a different factor VIII (FVIII) product did not develop anti-FVIII inhibitors nor did their immune profile change ...
Fitusiran Significantly Reduces Bleeding Events, Improves HRQoL in Hemophilia A, B
Once-monthly prophylaxis with the investigational small interference RNA therapeutic fitusiran was associated with a significant reduction in bleeding events when compared with clotting factor concentrate ...
STASEY Study Confirms Safety of Emicizumab Prophylaxis in Hemophilia A With FVIII Inhibitors
The safety profile of emicizumab prophylaxis was confirmed in a large, international population of patients with hemophilia A and factor VIII (FVIII) inhibitors ...
Low VWF Levels Linked With Lower CVD Risk
People with low levels of von Willebrand factor (VWF) had a lower risk of incident cardiovascular disease (CVD), according to a study published in ...
Efgartigimod Promotes Sustained Platelet Count Response in iTP
Treatment with efgartigimod (EFG) resulted in platelet count increases as well as high sustained platelet count responses in patients with persistent or chronic immune thrombocytopenia (iTP) ...
Emicizumab Can Be Used Without FVIII Inhibitors in Infants With Severe Hemophilia
Results of an interim analysis of the HAVEN 7 study indicated that emicizumab is safe and effective in infants with severe hemophilia A without factor VIII (FVIII) inhibitors ...
Study Reveals Approach to Testing Adolescent Girls for VWD
An adolescent who is being evaluated for acute heavy menstrual bleeding and has von Willebrand factor (VWF) antigen levels of 80% or lower on initial presentation should have the test repeated at a later timepoint when she is back to her normal state of health ...
TA More Effective, Affordable Than rVWF for VWD-Related Heavy Menstrual Bleeding
Tranexamic acid (TA) may be the more effective and affordable option for reducing heavy menstrual bleeding among women with von Willebrand disease (VWD) compared to recombinant von Willebrand factor (rVWF) ...
Small Study Suggests Subcutaneous Rondoraptivon Pegol Improves VWF/FVIII Levels in Type 2B VWD
Treatment with the pegylated aptamer rondoraptivon pegol significantly improved levels of von Willebrand factor (VWF)/factor VIII (FVIII), multimer patterns, and thrombocytopenia in patients with type 2B von Willebrand disease (VWD), according to findings from a small phase II study...
