Standard treatment for hemophilia B involves replacing the missing clotting factor so that the blood can clot properly. This is called factor 9 replacement therapy. Healthcare providers infuse the two main replacement therapies — plasma-derived factor concentrates and recombinant factor concentrates — into the bloodstream via a vein in the arm.
In both cases, patients receive infusions of the concentrates prophylactically, meaning they receive them regularly to prevent bleeding episodes. But if you have severe hemophilia B, you may also need to take a factor 9 concentrate before surgery or dental work.
People who have hemophilia usually learn to self-administer their replacement therapy at home. In fact the National Bleeding Disorders Foundation (NBDF) has self-infusion instructions for kids who are mature enough to learn how to infuse their own factor. The cost of factor 9 replacement therapy is significant. The average annual cost of clotting factor therapies for a person with severe hemophilia is roughly $300,000, and overall yearly medical expenses can be twice that.
Plasma-Derived Factor 9 Concentrates
Manufacturers derive plasma-derived factor 9 concentrates from human donations of blood or plasma.
The liquid part of blood, called plasma, contains clotting factors and other proteins, such as antibodies and albumin. Albumin is a protein made by your liver to help keep fluid from leaking out of your blood vessels.
All donated blood or plasma undergoes testing for viruses. The plasma goes through multiple processes to separate it into components, such as clotting factors. The clotting factors are freeze-dried and treated to kill (or make inactive) any viruses found.
Two plasma-derived factor 9 concentrates are available in the United States:
- AlphaNine SD contains only blood factor 9 and no other factors.
Profilnine contains factor 9 along with factor 2, factor 10, and low levels of factor 7.
Recombinant Factor 9 Concentrates
About three-fourths of people with hemophilia take recombinant factor 9 concentrates.
These products are developed in the lab using DNA technology. Recombinant factor 9 concentrates are not able to spread bloodborne viruses, because they are synthetic and not from humans.
Available recombinant factor 9 concentrates include:
- Alprolix
- Benefix
- Idelvion, which also contains albumin
- Ixinity
- Rebinyn
- Rixubis
The Medical and Scientific Advisory Council (MASAC) of the NBDF recommends recombinant factor 9 concentrates over plasma-derived concentrates because they are safer.
Read the full article here
