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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.


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.


Brand Name: Exjade, Jadenu
Treatment for: aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH)
An iron chelator, this drug is used to remove excess iron from the body.


Brand Name: Ferriprox
Treatment for:
An iron chelator, this drug is used to remove excess iron from the body. Iron overload (too much iron in the body) can occur in patients who get lots of red blood cell transfusions.


Brand Name: Soliris
Treatment for: paroxysmal nocturnal hemoglobinuria (PNH)
A drug approved by the U.S. Food and Drug Administration (FDA) and in Europe to treat paroxysmal nocturnal hemoglobinuria (PNH).


Brand Name: Promacta
Treatment for: aplastic anemia
Eltrombopag (Promacta) is used to increase the number of platelets (cells that help blood clot) and to decrease the risk of bleeding. It is a man-made platelet growth factor that signals your bone marrow to make more platelets. Eltrombopag was approved by the U.S. Food and Drug Administration in 2014 to treat aplastic anemia. It comes as a tablet and as a powder for oral suspension (liquid) to take by mouth.

Epoetin Alfa

Brand Name: Epogen, Procrit
Treatment for: aplastic anemia
A man-made version of a naturally occurring substance in the body (erythropoietin) that encourages the bone marrow to make more red blood cells.


Brand Name: G-CSF, Neupogen
Treatment for:
A man-made version of a naturally occurring substance in the body that promotes white blood cell growth in the bone marrow.


Brand Name: Fludara
Treatment for:
An antineoplastic agent, this medicine works by slowing or stopping the growth of cancer cells.


Brand Name: Tibsovo
Treatment for: acute myeloid leukemia (AML)
A small molecule inhibitor of isocitrate dehydrogenase-1 (IDH1), which is mutated in several forms of cancer, including acute myeloid leukemia (AML).


Brand Name: Revlimid
Treatment for: myelodysplastic syndromes (MDS)
An immunomodulatory and anti-angiogenic agent, this drug slows the growth of blood vessels feeding abnormal cells and kills abnormal 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.

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