Long-term prophylactic (preventive) treatment with a fixed dose of Esperoct (turoctocog alfa pegol) — approved in the U.S. and the EU in 2019 — was effective in preventing and stopping bleeding in people with severe hemophilia A, according to data from two Phase 3 clinical trials. Findings from…

Interim results of Phase 3 clinical studies investigating two major challenges in hemophilia — the development of inhibitors (antibodies to the drug used to treat bleeding episodes) and the need for repeated venous injection of blood clotting protein Factor VIII (FVIII) — were recently released. The results were presented by…

Hemophilia is a blood disorder that’s usually genetically inherited from parents. People born with hemophilia have little or no clotting factor – a protein needed for normal blood clotting. Hemophilia can also be acquired, meaning patients don’t inherit the disease from their parents but instead develop it during their lifetime. This…

Researchers have identified four distinct subgroups of hemophilia A patients according to their profile of factor VIII-targeting antibodies. This may help predict those at risk for developing inhibitors that halt the efficacy of FVIII replacement therapy. The results were presented in a poster titled “Data Coming out of the…

When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and effectively lowered the number of bleeding episodes in patients with severe hemophilia A, according to data from a Phase 1 clinical trial and its long-term extension study. Importantly, reductions in the number of bleeds…

A population-based pharmacokinetic approach for tailored treatment regimens of hemophilia A patients may contribute to an increase in treatment adherence and a decrease in annual bleeding episodes, a study shows. The study, “Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing,” was published…

Some hemophilia patients may, over time, develop inhibitors which restrict the efficacy of treatment making bleeding episodes more difficult to stop. MORE: Intracranial hemorrhage risk – when to see a doctor According to the Centers for Disease Control and Prevention (CDC), although the majority of people living with…

Low-dose recombinant factor VIII lowered bleeding rates in Chinese children with hemophilia, and the earlier the preventive treatment began, the more effective it was in minimizing bleeding episodes, according to a new study. The research, “The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A:…

The levels of sclerostin, a protein that regulates bone formation, may be linked to disease severity and reflect bone alterations in children with hemophilia A, a study suggests. The study, “Serum Sclerostin Level and Bone Mineral Density in Pediatric Hemophilic Arthropathy,” was published in The Indian…