Treatment

Treatment Regimen and Hemophilia Severity

The majority of male Registry participants with mild or moderate hemophilia A are on an episodic (on-demand) treatment regimen, in which they receive treatment only in response to a bleeding episode (91% and 62%, respectively). Among participants with mild or moderate hemophilia A, continuous prophylaxis is the second most common treatment regimen (4% and 32%, respectively). In contrast, among participants with severe hemophilia A, continuous prophylaxis is the most common treatment regimen, with 77% of participants using this method, followed by episodic or event-based treatment (17%). Among participants with mild, moderate, or severe hemophilia A, event-based, intermittent or short-term prohylaxis, was used by 5%, 6%, and 4%, respectively (Figure 28).

The majority of participants with mild or moderate hemophilia B are on an episodic treatment regimen (91% and 80%, respectively). Continuous prophylaxis is the second most common treatment regimen among participants with mild or moderate hemophilia B (5% and 16%, respectively). Among the male participants with severe hemophilia B, continuous prophylaxis is the most common treatment regimen, followed by episodic or event-based treatment (69% and 25%, respectively). Among participants with hemophilia B, approximately 4% of mild and moderate and 5% of severe used event-based prophylaxis, intermittent or short-term prophylaxis (Figure 29).

Figure 28. Treatment regimen for male Registry participants with hemophilia A, by severity
Figure 28. Treatment regimen for male Registry participants with hemophilia A, by severity

Figure 28. Treatment regimen for male Registry participants with hemophilia A, by severity
Mild Moderate Severe
Episodic (on-demand) 1517 90.7% 776 61.5% 711 16.6%
Immune tolerance induction (ITI) without continuous prophylaxis with a by-passing agent * * 11 0.9% 87 2.0%
Prophylaxis – Continuous 73 4.4% 397 31.5% 3289 76.6%
Prophylaxis – Continuous with by-passing agents plus Immune tolerance induction (ITI) * * * * 59 1.4%
Prophylaxis – Event-based, short-term or intermittent 78 4.7% 74 5.9% 150 3.5%

* Counts less than six have been suppressed to protect patient confidentiality

Figure 29. Treatment regimen for male Registry participants with hemophilia B, by severity
   Figure 29. Treatment regimen for male Registry participants with hemophilia B, by severity

Figure 28. Treatment regimen for male Registry participants with hemophilia A, by severity
Mild Moderate Severe
Episodic (on-demand) 392 90.5% 572 80.3% 187 25.2%
Immune tolerance induction (ITI) without continuous prophylaxis with a by-passing agent * * * * * *
Prophylaxis – Continuous 22 5.1% 111 15.6% 511 69.0%
Prophylaxis – Continuous with by-passing agents plus Immune tolerance induction (ITI) * * * * * *
Prophylaxis – Event-based, short-term or intermittent 19 4.4% 27 3.8% 37 5.0%

* Counts less than six have been suppressed to protect patient confidentiality

Treatment Products and Age

Recombinant clotting factor concentrates were the most commonly used medication, regardless of age group; also common among the younger age groups were non-plasma products.

Figure 30. Product usage among male Registry participants with hemophilia, by age
Figure 30. Product usage among male Registry participants with hemophilia, by age

Figure 30. Product usage among male Registry participants with hemophilia, by age
<2 years 2–10 years 11–19 years 20–44 years 45–64 years 65+ years
Non-Blood Products 61 16.5% 539 25.2% 472 21.4% 308 10.6% 128 10.7% 42 12.0%
No Products 81 21.9% 172 8.0% 146 6.6% 157 5.4% 134 11.2% 56 16.0%
Other Products 10 2.7% 72 3.4% 73 3.3% 138 4.7% 50 4.2% 16 4.6%
Plasma Derived Clotting Factor Concentrates 43 11.6% 191 8.9% 110 5.0% 279 9.6% 170 14.2% 39 11.2%
Blood Products 12 3.2% 10 0.5% * * * * 6 0.5% * *
Recombinant Clotting Factor Concentrates 250 67.6% 1792 83.8% 1917 87.0% 2471 84.7% 872 73.0% 243 69.6%

* Counts less than six have been suppressed to protect patient confidentiality