The goal of hemophilia treatment is rapid and effective replacement of the missing clotting factor and prevention of complications such as joint damage. Clotting factor dosing is determined by a variety of things, including the patient’s weight, disease severity, bleeding patterns and comorbidities. Depending on severity, factor levels in the blood and bleeding triggers, treatments are either on-demand (episodic) or ongoing clotting factor replacement to prevent bleeding (prophylaxis).
- Treatment of bleeds after they occur (required to stop bleeds after they start)
- Even one bad bleed can lead to permanent debilitating pain or can be life threatening if the brain or a major organ is involved
- Challenge: Episodic treatment does not prevent bleeds
Prophylaxis (clotting factor replacement)
- Standard of care since the 1990s
- Regular administration of clotting factor to prevent bleeds
- The goal of this therapy is protection from traumatic and unexpected bleeds and prevention of joint damage and joint disease
- Challenge: Patient adherence with long-term prophylactic therapy
Both episodic and prophylaxis clotting factor can be billed under either the medical or prescription drug benefit. If it is acquired through a home infusion company, it is billed under the medical benefit.
Bleeding Episodes & Inhibitors
For 20% - 35% of people with hemophilia, treating bleeding episodes can be extremely difficult. This is due to the development of an antibody, called an inhibitor, that stops the clotting factor from doing its job, rendering the standard treatment ineffective. Bleeding is more difficult to control and can lead to joint disease and disability. Inhibitor development is the most serious complication of factor replacement therapy and is very costly and complex to manage.