The Evolution of the Hemophilia Treatment Landscape

Robert F. Sidonio Jr, MD, assistant professor of pediatrics at Emory University, Children's Healthcare of Atlanta, gives an overview of the different hemophilia therapies and recent improvements in the treatment landscape. 


So the treatment landscape up until recently hadn’t changed much. They started off in the 60s with cryoprecipitate and then they were able to do factor concentrates in the 70s and 80s. That was a huge improvement for patients; they could actually treat themselves at home and not have to come to the hospital. And then of course the 1980s came, HIV and hepatitis devastated the community. And then there was a push to develop recombinant products, so the ones that were not developed from human plasma.
So there’s been an evolution but there really hasn’t been any significant change up until recently. They’ve extended the half-life of the products so it lasts a little longer in the body. But all the therapies up until just recently have been through IV therapies. So that means you would have to start an IV with a butterfly needle to get the therapy in. And as anybody knows, young children don’t like needles, that hasn’t changed. And so the new therapies are a different route. Subcutaneous is obviously a preferred route for some of these new therapies. Emicizumab particularly is a subcutaneous drug so that’s really changed the landscape. It’s only for hemophilia A.

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