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.
Filgrastim
Brand Name: 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.
Fludarabine
Brand Name: Fludara
An antineoplastic agent, this medicine works by slowing or stopping the growth of cancer cells.
Heparin
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.
Hydroxyurea
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.
Iptacopan
Brand Name: Fabhalta
FABHALTA, a complement factor B inhibitor, is the first oral medication approved to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). It was approved by the U.S. Food and Drug Administration in December 2023. Fabhalta is taken twice a day in a capsule form.
Ivosidenib
Brand Name: Tibsovo
A small molecule inhibitor of isocitrate dehydrogenase-1 (IDH1), which is mutated in several forms of cancer, including acute myeloid leukemia (AML).
Lenalidomide
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.
Luspatercept-aamt
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.
Mycophenolate Mofetil
Brand Name: CellCept
A drug that lowers the body's immune response, also referred to as an immunosuppressant drug.
Pegcetacoplan
Brand Name: EMPAVELI
EMPAVELI® is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is given by infusion under the skin (subcutaneously) into your stomach (abdomen), back of upper arms, hips, or thighs using an infusion pump.