What is active treatment or active drug therapy?

Doctors will sometimes refer to certain drugs as “active treatment” or “active drug therapy.” Active treatments are drugs used to treat the underlying cause of the bone marrow failure. These drugs are typically approved by the U.S. Food and Drug Administration (FDA) to treat a specific disease. It is important to note that active treatments do not cure the disease. The only cure for a bone marrow failure disease is a bone marrow/stem cell transplant.

For aplastic anemia, antithymocyte globulin (ATG), typically given with cyclosporine, is an active drug therapy often used. Learn more about treatments for aplastic anemia.

For MDS, there are three FDA approved active treatments currently available. These include lenalidomide (Revlimid®) decitibine (Dacogen®) and azacitidine (Vidaza®). Learn more about treatments for MDS.

For PNH patients the drug eculizumab (Soliris®) is the only FDA approved treatment currently available. Learn more about treatments for PNH.