Drugs

More drugs are available to treat bone marrow failure disease than ever before. Researchers constantly look for ways to enhance drugs already on the market and develop new ones with fewer side effects. Some drugs treat only one type of the disease, while others may be used to treat more than one type. Check with your doctor to find out which drugs are right for you and why.

Alemtuzumab

Brand Name: Campath
Treatment for: aplastic anemia
A monocolonal antibody - a medicine that is engineered to look for a specific substance in the body. Alemtuzumab attaches to and kills white blood cells called lymphocytes. In certain types of aplastic anemia, lymphocytes are responsible for attacking the bone marrow stem cells. Alemtuzumab is in clinical trials for treating aplastic anemia. It is approved by the U.S. Food and Drug Administration (FDA) for treating certain types of leukemia and is helpful in treating other conditions that require immunosuppressive therapy.

Antithymocyte Globulin

Brand Name: ATG, Atgam, Thymoglobulin
Treatment for: aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH)
ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH.

Arsenic Trioxide

Brand Name: Trisenox, ATO
Treatment for: myelodysplastic syndromes (MDS)
An antineoplastic agent, this medicine works by slowing or stopping the growth of cancer cells.

Azacitidine

Brand Name: 5-azacytidine, Vidaza
Treatment for: myelodysplastic syndromes (MDS)
A demethylating agent, this medicine interacts with DNA to restore normal growth of blood cells in the bone marrow.

Coversin

Brand Name:
Treatment for: paroxysmal nocturnal hemoglobinuria (PNH)

Cyclophosphamide

Brand Name: Cytoxan
Treatment for: aplastic anemia
An alkylating agent, a class of chemotherapy drugs which interfers with the growth of diseased cells. Cyclophosphamide is approved by the U.S. Food and Drug Administration (FDA) for treating certain diseases. It is still an investigational therapy, in combination with other medications, for treating aplastic anemia and other forms of bone marrow failure.

Cyclosporine

Brand Name: Neoral, Sandimmune
Treatment for: aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH)
An immunosuppressant, this drug that lowers the body's immune response.

Daclizumab

Brand Name: Zenapax
An immunosuppressant, this drug lowers the body's immune response.

Darbepoetin Alfa

Brand Name: Aranesp
Treatment for: aplastic anemia
A growth factor (cytokine), it is a man-made version of a naturally occurring substance in the body, called erythropoietin, that encourages the bone marrow to make more red blood cells.

Decitabine

Brand Name: Dacogen
Treatment for: myelodysplastic syndromes (MDS)
A demethylating agent, this drug interacts with DNA to restore normal growth of blood cells in the bone marrow.

AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any drug, treatment or therapy listed on this website. Some therapies listed on our site are considered experimental for the treatment of bone marrow failure diseases. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding any therapy, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.