Prophylactic medication or therapy is used to prevent a disease from occurring. Prophylaxis has been the standard of care since the 1990’s for moderate to severe patients with insufficient clotting factor in their blood who are susceptible to frequent bleeding episodes. The goal of regular and ongoing treatment is to prevent bleeds and minimize risk to major organs and/or permanent joint damage. There are two types of prophylaxis for prevention:
Clotting Factor Replacement Therapy
- Delivered through intravenous infusion (generally self-infused in the home)
- Average two to three doses weekly
- Challenges: Long-term patient adherence to the therapy; potential issues accessing veins can sometimes lead to direct vein implantation (PORT) for infants and small children which can increase risk of infection
- Delivered through subcutaneous injection (generally self-administered in the home)
- Weekly, every two or four weeks dosing options; uses same amount of drug per 4 weeks regardless of frequency
- Challenge: Clotting factor replacement therapy is still needed to treat bleeds, if and when they occur; potential for a drug interaction with some bypassing agents
Both episodic and prophylaxis therapies can be billed under either the medical or pharmacy benefit.
Bleeding Episodes & Inhibitors
People with hemophilia have a better quality of life today than ever before, but medical complications can still occur. Developing an inhibitor is one of the most serious complications of a bleeding disorder. Inhibitors occur when the body develops antibodies (proteins) to the infused clotting factor treatments. The antibodies are seen as a foreign substance needing to be destroyed, rendering the standard treatment ineffective. This results in increased morbidity and mortality and has the potential to substantially increase the financial burden for people with hemophilia.