When to start and stop
There are still varying opinions regarding the best time to start and stop prophylaxis.
The most cost-effective approach is to start prophylaxis early in order to preserve normal joints. Prophylaxis has been the standard of care in many European countries for more than 50 years, however the associated high cost has hampered its wide-scale adoption.
Access to large quantities of clotting factor concentrates needed for prophylaxis is certainly an important factor in determining when to start treatment and how long it should be continued.
In countries where treatment is readily available, most agree that prophylaxis should be started before irreversible joint damage has occurred. Some doctors believe it should begin after the first joint bleed, or by a certain age (typically two or three years old). Others recommend waiting until two or more bleeds have occurred.
Where access to clotting factor concentrates is limited, lower doses of prophylaxis given more frequently may be an effective option. To allow greater access to prophylaxis, cost-efficacy studies are needed to determine the minimum effective dosage, thereby maximizing treatment product for the greatest number of patients.
It is also unclear whether all patients should remain on prophylaxis indefinitely as they become adults. Although some studies suggest that young adults can do well without prophylaxis, more studies are needed before a clear recommendation can be made. Patients often decide for themselves whether to continue prophylaxis or not.
Updated December 2014