Although no cure for hemophilia is currently available, a number of therapies can be used to control bleeding and other symptoms. These treatments can substantially increase life expectancy by lowering the risk of life-threatening complications such as bleeding in the brain, especially in people with severe hemophilia.
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The development of neutralizing antibodies, or inhibitors, against the clotting factor in replacement therapies administered to people with hemophilia may depend on the type of product used, a recent study has found. In previously untreated people with severe hemophilia A, or those who lack…
Treatment with Octapharma‘s Nuwiq is associated with a low risk of developing inhibitors — antibodies that prevent the medication from working properly — in previously untreated patients (PUPs) with hemophilia A, according to the results of a clinical trial. These findings were published in the journal Thrombosis…
The ongoing Phase 1/2 Alta trial continues to show that a single infusion of SB-525, an investigational gene therapy, leads to stable and clinically relevant improvements in clotting factor VIII and marked reductions in the need for replacement therapy in patients with hemophilia A. This preliminary follow-up data…
A newly designed platform based on the principle of “reverse vaccination” could be used to prevent the development of an immune response that blocks the effectiveness of treatments for hemophilia and other disorders. “For nearly a third of patients with hemophilia A … their own immune system is…
Patients with hemophilia A who switched to Kovaltry (octocog alfa) — a recombinant, or lab-made, factor VIII (rFVIII) replacement therapy given at home — had significantly fewer bleeds than with their previous standard half-life replacement therapies, a real-world study in Italy reported. The therapy also reduced the frequency…
For some patients with moderate or severe hemophilia, switching from standard to extended half-life replacement therapy results in improvements in key outcome measures, a study in Canada found. Most of these self-reported gains in health-related quality of life, and physical, mental, and social functioning three months after the switch,…
Mononine, a long-standing treatment to prevent and control bleeding in hemophilia B, will be discontinued due to declining demand in recent years. CSL Behring, the therapy’s manufacturer, said the decision was based on the emergence of next-generation hemophilia B treatments now in greater use. “Over time,…
Adults and children with severe hemophilia A who are receiving Nuwiq (recombinant human coagulation factor VIII) as their first replacement therapy have a similar risk of developing inhibitors as those treated with plasma-derived therapies containing von Willebrand factor, the final results of the NuProtect trial show.