Margaret Ragni, MD, MPH: Women who are Hemophilia Carriers Need Treatment Options

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While this population was previously thought to be unaffected, studies within the last few years have shown that women with hemophilia experience joint and bleeding problems.

The once-weekly antihemophilic factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl (ALTUVIIIO) treatment for hemophilia A (previously known as efanesoctocog alfa) gives factor VIII “Superman” qualities for an entire week after treatment, expert Margaret Ragni, MD, MPH, Professor of Medicine and Clinical Translational Science, and Medical Director, Hemophilia Center of Western Pennsylvania, said in an interview with HCPLive.

“The minute von Willebrand factor is no longer in circulation after a couple days, factor VIII isn’t either,” she explained. “What this does is it binds the von Willebrand factor and factor VIII together so it continues in the circulation protected and not broken down.”

The antihemophillic factor VIII therapy recently approved by the US Food and Drug Administration for adults and children with hemophilia A only requires treatment once per week, reducing the treatment burden for patients when compared with others on the market.

Now that the technology has advanced to allow this treatment option for patients, Ragni stated that more research needs to be focused on treating women who are hemophilia carriers because saying that they’re often overlooked would be an “understatement”.

While this population was previously thought to be unaffected, studies within the last few years have shown that women who carry hemophilia experience joint and bleeding problems that can coincide with menstrual bleeding.

“One of the questions with these different drugs, including this efa (ALTUVIIIO), that are coming up are ‘well, can't we look at these in the women and see if they might–on a once a week basis during heavy menses–make a huge difference?’” she said.

Watch more of HCPLive's interview with Dr. Margaret Ragni.

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