A 48-year-old man with severe hemophilia A developed a rare intramural hematoma with blood leaking into one of the layers of the intestine wall that was successfully treated with an early infusion of the missing blood clotting factor, a recent case report described. Prophylactic treatment prevented its recurrence. A…

Female hemophilia A  patients and carriers have more frequent bleeding than males, yet a much smaller proportion receive replacement therapy to prevent or stop them, a U.S. study finds. The medical and pharmacy costs for women with hemophilia A or carriers were up to about 14 times lower…

MRI screening can identify subclinical joint bleeding in some patients with severe hemophilia A on preventive treatment, according to a study in the Netherlands. Subclinical bleeds occur without symptoms and usually cannot be detected by standard physical and ultrasound examinations. “These MRI findings support the hypothesis that subclinical…

The U.S. Food and Drug Administration (FDA) is extending its review of BioMarin Pharmaceutical’s request to approve the gene therapy Roctavian (valoctocogene roxaparvovec) for adults with severe hemophilia A. A decision had been expected by the end of this month, but that date has now been…

Most children with hemophilia A given Altuviiio (efanesoctocog alfa) once a week were bleed-free throughout about a year of treatment. That’s according to top-line data from the Phase 3 XTEND-Kids trial (NCT04759131) announced by Sobi and Sanofi, the therapy’s co-developers, less than a month after…

Note: This story was updated Feb. 24, 2023, to correct the secondary headline since there are several other recombinant factor VIII therapies available.  Altuviiio is a first-in-class therapy, but not the first. Efanesoctocog alfa, Sobi and Sanofi’s first-in-class, long-lasting recombinant or man-made factor…

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…

As a person with von Willebrand disease (VWD) and hemophilia, bruises have been a near-constant presence in my life. VWD and hemophilia are bleeding disorders that affect the body’s ability to form blood clots and stop bleeding. Both can cause spontaneous and excessive bleeding, including bruising, heavy…

The European Commission has approved Hemlibra (emicizumab) as a routine preventive treatment for people with moderate hemophilia A without inhibitors. “We welcome the European Commission’s decision to approve Hemlibra also for people with moderate hemophilia A in the EU,” said Levi Garraway, MD, PhD, Roche’s chief medical officer…

High levels of antibodies against clotting factor VIII (FVIII) were associated with a slower response to immunosuppressive treatments in a small group of people with acquired hemophilia A in new study in China. Of 34 patients, 29 achieved a complete response to their first-line immunosuppressive therapy. Still, a lower…