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MDS Drugs and Treatments

More drugs and treatments 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: Calciparine, Liquaemin
Heparin is in a class of medications called anticoagulants ('blood thinners'). It works by decreasing the clotting ability of the blood. Heparin is also used to stop the growth of clots that have already formed in the blood vessels, but it cannot be used to decrease the size of clots that have already formed. 


Brand name: Hydrea
Hydroxyurea is in a class of medications called antimetabolites. Hydroxyurea treats cancer by slowing or stopping the growth of cancer cells in your body. It can be used alone or with other medications to treat a certain types of leukemias. 

Iron Chelation

Iron chelation therapy is the main treatment used when you have a condition called iron overload. Iron overload means you have too much iron in your body. This can be a problem for people who get lots of red blood cell transfusions.


Brand name: 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.


Brand name: REBLOZYL®
First erythroid maturation agent (EMA) approved by the FDA for the treatment of anemia in adult patients with myelodysplastic syndromes (MDS) who have been failed an erythropoiesis stimulating agent (ESA) or are unlikely to respond to an ESA, and are requiring transfusions of 2 or more red blood cell (RBC) units over 8 weeks.


Brand name: Rayos, Sterapred, Deltasone
Prednisone a man-made version of a hormone produced naturally by the adrenal gland that can help reduce inflammation and stop allergic responses. It is prescribed when the body is not producing enough of this chemical on its own.


Brand name: NPlate
Romiplostim is in a class of medications called thrombopoietin receptor agonists. It works by causing the cells in the bone marrow to produce more platelets. Romiplostim injection is used to increase the number of platelets enough to lower the risk of bleeding, but it is not used to increase the number of platelets to a normal level.


Sabatolimab (MBG453) is a novel immuno-myeloid therapy that binds to TIM-3 on immune cells, facilitating antileukemic immune activation and phagocytic killing of leukemic cells. Sabatolimab also binds to TIM-3 on leukemic cells, potentially impeding self-renewal of LSCs via inhibition of TIM3-galectin-9.


Brand name: Thalomid
Thalidomide is an old drug, though it has not been widely available since the 1960s, when it was found to cause birth defects.


Brand name: Venclexta
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is used alone or in combination with other cancer medicines to treat these conditions.