Glossary of Drug Names

The following is a glossary of drug names used in the treatment of bone marrow failure diseases. You can also browse the glossary of terms.
Click one of the letters above to advance the page to terms beginning with that letter.

A

alemtuzumab »

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(Campath) 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 other conditions that require immunosuppressive therapy.

amifostine »

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(Ethyol) A cytoprotective agent or chemoprotectant. This drug is approved by the U.S. Food and Drug Administration (FDA) for use during cancer therapy to protect the body's normal cells. Amifostine is being studied in clinical trials, as a single agent or in combination with other medicines, for treating myelodysplastic syndromes.

antithymocyte globulin »

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(ATG, Atgam, Thymoglobulin) ATG is an immunosuppressant, a drug that lowers the body's immune response. Scientists believe that aplastic anemia happens when the immune system attacks and destroys bone marrow stem cells. ATG kills the specific cells that are attacking the bone marrow stem cells. This allows the bone marrow to grow and make new blood cells. ATG may be used to treat other bone marrow failure diseases in some cases. ATG is approved by the U.S. Food and Drug Administration (FDA) for treating moderate and severe aplastic anemia. ATG is commonly used with another drug called cyclosporine.

arsenic trioxide »

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(Trisenox, ATO) An antineoplastic agent, this medicine works by slowing or stopping the growth of cancer cells. It is approved by the U.S. Food and Drug Administration (FDA) for treating acute promylocitic leukemia (APL). Currently, researchers are studying arsenic trioxide, as a single agent or in combination with other medicines, for treating myelodysplastic syndromes.

azacitidine »

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(5-azacytidine, Vidaza) A demethylating agent, this medicine interacts with DNA to restore normal growth of blood cells in the bone marrow. It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Azacitidine is approved by the U.S. Food and Drug Administration (FDA) for treating all myelodysplastic syndromes (MDS) subtypes.

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C

cyclophosphamide »

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(Cytoxan) 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

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(Neoral, Sandimmune) An immunosuppressant, this drug that lowers the body's immune response. Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure.

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D

daclizumab

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(anti-CD25, Zenapax) An immunosuppressant, this drug lowers the body's immune response. It is approved by the U.S. Food and Drug Administration (FDA) for use in transplant patients to reduce the chance of organ rejection. Currently, researchers are studying daclizumab for treating aplastic anemia to determine whether it will be effective and safe to use.

darbepoetin alfa

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(Aranesp) 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. It is in a class of medicines called erythropoiesis-stimulating agents (ESAs). Darbepoetin alfa can help improve red blood cell counts in bone marrow failure patients whose natural erythropoietin levels are low. It is given by injection under the skin (subcutaneous) or in the vein (intravenous). Darbepoetin alfa is approved by the U.S. Food and Drug Administration (FDA) for treating anemia.

decitabine

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(Dacogen) A demethylating agent, this drug interacts with DNA to restore normal growth of blood cells in the bone marrow. It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Decitabine is approved by the U.S. Food and Drug Administration (FDA) for treating all MDS subtypes, though it is used most commonly to treat higher-risk MDS.

deferasirox

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(Exjade) An iron chelator, this drug is used to remove excess iron from the body. It comes in a tablet form which is dissolved in juice or water and taken by mouth. Iron build-up can occur in patients who get a lot of red blood cell transfusions. Deferasirox binds with the iron in the body and is removed by the kidneys. Bone marrow failure disease patients are at risk for developing iron overload if they have had more than 20 units of packed red blood cells.

deferiprone

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(Ferriprox) 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. Deferiprone comes in a pill form and is taken by mouth. Deferiprone is approved for use in Europe. Researchers in the U.S. are studying deferiprone for treating iron overload in patients with thalassemia. Deferiprone binds with iron in the bloodstream and is removed from the body by the kidneys. Bone marrow failure patients are at risk for developing iron overload if they have had more than 20 units of packed red blood cells.

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E

eculizumab

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(Soliris) A drug approved by the U.S. Food and Drug Administration (FDA) and in Europe to treat paroxysmal nocturnal hemoglobinuria (PNH). Eculizumab causes the complement system, a part of your immune system, to be less active. By doing this it reduces hemolysis, the destruction of red blood cells, in patients with PNH. It is in a class of drugs call monoclonal antibodies.

epoetin alfa

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(Epogen, Procrit) A man-made version of a naturally occurring substance in the body (erythropoietin) that encourages the bone marrow to make more red blood cells. Epoetin alfa can help improve red blood cell counts in bone marrow failure disease patients whose natural erythropoietin levels are low. It is given by injection under the skin (subcutaneous) or in the vein (intravenous). Epoetin alfa is approved by the U.S. Food and Drug Administration (FDA) for treating anemia. It is in a class of drugs called growth factors (cytokines).

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F

filgrastim

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(G-CSF, Neupogen) A man-made version of a naturally occurring substance in the body that promotes white blood cell growth in the bone marrow. It is known as a hematopoietic growth factor or colony stimulating factorand is given by injection under the skin (subcutaneous) or in the vein (intravenous). Filgrastim is approved by the U.S. Food and Drug Administration (FDA) for use in cancer patients who are at risk of developing infection due to cancer treatment. It is also approved for treating other patients who have a very low white blood cell count. It is in a class of drugs called growth factors (cytokines).

fludarabine

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(Fludara) An antineoplastic agent, this medicine works by slowing or stopping the growth of cancer cells. The U.S. Food and Drug Administration (FDA) approved fludarabine for treating chronic lymphocytic leukemia (CCL). Researchers are studying fludarabine in combination with other medicines for treating bone marrow failure.

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L

lenalidomide

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(Revlimid) 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. Lenalidomide is a capsule that is taken by mouth. It is approved for treating low-risk, transfusion-dependent myelodysplastic syndrome (MDS) patients with an abnormality of chromosome 5q. It is currently in clinical trials to test its efficacy with a broader range of MDS patients. For some MDS patients, lenalidomide can improve anemia and reduce or eliminate the need for blood transfusions.

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M

mycophenolate mofetil (CellCept)

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A drug that lowers the body's immune response, also referred to as an immunosuppressantdrug. Mycophenolate mofetil is approved by the U.S. Food and Drug Administration (FDA) to prevent organ rejection in people who have received a kidney, heart, or liver transplant. This medicine comes as a capsule, tablet, or liquid that is taken by mouth. Researchers are currently studying MMF in combination with other medicines for treating aplastic anemia.

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T

thalidomide

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(Thalomid) Thalidomide is an old drug, though it has not been widely available since the 1960s, when it was found to cause birth defects. Currently it is approved by the U.S. Food and Drug Administration (FDA) for use in treating a skin disease associated with leprosy.

Thalidomide is being studied, either as a single agent or in combination with other medicines, for treating of myelodysplastic syndromes (MDS). These clinical trials are studying the effectiveness and safety of the drug. A pilot study of thalidomide, published in 2001, found the drug improved blood counts in some patients and enabled others to become transfusion-independent. Thalidomide has some severe side effects which may limit its application as a treatment for MDS.

TLK-199

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TLK-199 is a small molecule drug that stimulates the bone marrow to produce blood cells. Currently TLK-199 is in Phase II of the Food and Drug Administration's (FDA's) clinical trial for the treatment of myelodsyplasitc syndromes (MDS). www.clinicaltrials.gov

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W

warfarin

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(Coumadin) A blood thinner, or anticoagulant. It is used to prevent blood clots from forming. Warfarin is a tablet that is taken by mouth.

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