Several different types of medications are available to treat hemophilia and other bleeding disorders. For patients with moderate to severe hemophilia, treatment regimens focus on replacing the missing clotting factor to stop and prevent bleeding episodes. Other drug therapies focus on increasing the body’s ability to produce factor and on relieving disease symptoms as they arise.
Recombinant Versus Plasma-Derived Clotting Factor
Plasma-derived clotting factor replacement treatments use concentrated factor extracted from human plasma. During the 1980s, there was no way to screen donated blood for HIV and as many as 90% of patients with severe hemophilia contracted HIV. Today, there are significant safety measures in place to prevent the transmission of bloodborne viruses from plasma-derived factor. No cases of HIV contracted through clotting factor replacement treatments have been reported in over 20 years.
In the early 1990s, genetically-engineered (recombinant) factor concentrates were approved by the FDA as an alternative to plasma-derived factor. Since recombinant factors do not come from human blood, they cannot transmit bloodborne viruses.
Treating Factor Inhibitors
Approximately 20% of patients with hemophilia A and 3% of patients with hemophilia B develop inhibitors, antibodies produced by the immune system to attack infused factor before it can stop a bleeding episode. Inhibitors present an additional hurdle to treating hemophilia because the immune response must be neutralized for factor infusions to be effective. For patients with low amounts of inhibitors in their blood, infusing higher doses of factor may be enough to overcome inhibitors.
Patients with high levels of inhibitors may successfully train their bodies to tolerate factor and not produce antibodies. This approach is called Immune Tolerance Therapy (ITT) and involves regular infusions of factor (as often as every day). ITT works for roughly 60%-80% of hemophilia patients with inhibitors.
Current Hemophilia Treatment Options
Recombinant coagulation/antihemophilic factor for hemophilia A to replace factor VIII
- Helixate FS
- Kogenate FS
- NovoSeven RT
Plasma-derived coagulation/antihemophilic factor for hemophilia A to replace factor VIII
Recombinant coagulation/antihemophilic factor for hemophilia B to replace factor IX
Plasma-derived coagulation/antihemophilic factor for hemophilia B to replace factor IX
Monoclonal antibodies to prevent bleeding episodes for hemophilia A patients with inhibitors
- Emicizumab (Hemlibra)
Hormone therapy to stimulate factor VIII or factor IX production
Antifibrinolytics to stabilize clots
Topical treatments for external injuries
- Fibrin sealant (Tisseel VHSD)
Pain management for treating damaged joints and other disease symptoms
- Acetaminophen (Tylenol)
- Opioids (oxycodone, morphine, codeine, methadone)
Vaccinations for diseases transmitted by human blood
Hemophilia Treatments in Development
A number of pharmaceutical companies are developing treatments to combat hemophilia symptoms, increase time between prophylactic infusions, and more effectively manage disease complications. Some treatments under development even aim to cure hemophilia.
Here is an overview of different hemophilia treatments in development.
The effects of these hemophilia treatments are designed to last longer than their traditional counterparts, reducing the frequency of infusions.
- CSL Behring: Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689)
- OPKO Health: Factor VIIa-CTP
- Novo Nordisk: Turoctocog alfa pegol (N8-GP)
- Bayer: BAY 94-9027
More Effective Treatments
Some new hemophilia products are aimed at increasing the efficacy and/or potency of existing treatments. These treatments may be especially important for people with inhibitors for whom traditional hemophilia treatments are no longer effective.
Gene Therapy for Hemophilia
Gene therapy treatments focus on curing hemophilia by replacing or deactivating the faulty gene causing the disease. While gene therapy is still in its infancy, several clinical trials for hemophilia treatments are showing positive results.
- BioMarin Pharmaceutical: BMN 270
- Spark Therapeutics: SPK-9001
- uniQure: AMT‐060, AMT-061
- Sangamo Biosciences: SB-FIX ZFN
- Sangamo Biosciences: SB-525
Contact NCHS to learn how we can assist you through a medication change and about other support services we offer.
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