Small vesicles in the saliva of people with severe hemophilia A may explain why these patients rarely have bleeds in their mouth and throat. The vesicles contain a protein complex that’s able to induce blood coagulation in these patients, thereby reducing bleeding. The findings may help “develop innovative approaches…
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HAVEN 3 Data Supports Use of Hemlibra to Treat Hemophilia A Patients Without Factor VIII Inhibitors
Administration of once-weekly Hemlibra (emicizumab-kxwh) significantly reduced bleeding rates in patients with Hemophilia A compared to other clotting factor VIII prophylaxis (preventive) therapy, according to the latest results of the Phase 3 HAVEN 3 study announced by Roche. Developed by Chugai Pharmaceutical, Roche, and Genentech,…
Hemophilia A, the most common type of hemophilia, is a bleeding disorder that is inherited in an X-linked, recessive manner. This is because the F8 gene, which can cause hemophilia A when mutated, sits on the X chromosome. In addition, because hemophilia A is a recessive condition, it…
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High Adherence to Prophylaxis Improves Physical Activity of Patients With Hemophilia A, Study Says
High adherence to prophylactic treatment not only reduces the number of spontaneous bleeding episodes in patients with hemophilia A, but it also improves their physical activity, according to a study. The study, “Physical activity improved by adherence to prophylaxis in an Italian population of children, adolescents and…
A person in Germany with severe hemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), the first time the gene therapy has been used commercially in Europe, according to BioMarin Pharmaceutical, its developer. “Today represents an important milestone for the hemophilia community and for patients and…
Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy approved for adults with severe hemophilia A, is expected to be widely available in the U.S. this month, according to its developer, BioMarin. BioMarin also is working with authorities in Germany, Italy, and France to secure market access to…
Preventive treatment with low-dose Hemlibra (emicizumab-KXWH) significantly reduced bleeding and improved quality of life in young children with hemophilia A, regardless of inhibitor status, a real-world study in China found. But ultrasound scans showed signs of worsening joint health despite fewer clinically apparent bleeds, suggesting that lower doses may not…
Physical activities with a high risk of a collision are linked with a greater risk of bleeding in people with moderately severe hemophilia A, a six-month U.S. study reports. This risk was higher for people using on-demand versus preventive replacement factor VIII (FVIII) therapies, the researchers found, and they suggested…
Clinical trials of Hemlibra (emicizumab-kxwh) continue to show significant reductions in bleeding episodes in patients with hemophilia A with inhibitors, Genentech recently announced. Data from Hemlibra’s clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. The results show that in hemophilia A patients with inhibitors…
Teens and young adults with moderate and severe hemophilia A on continuous preventive treatment show levels of physical activity similar to young people without the disease, but less than half of teenagers met the weekly physical activity levels recommended by the World Health organization (WHO), a study reports. “We…