Aplastic Anemia 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.
Growth Factors
Growth factors
Growth factors:
Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts.
Red…
are naturally occurring hormones in your body that signal your
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
to make more of certain types of blood cells. Man-made growth factors may be given to some people with
bone marrow failure
bone marrow failure:
A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be…
diseases to help increase
red blood cell
red blood cell:
The most numerous type of blood cell in healthy people. Red blood cells contain hemoglobin, a protein that picks up oxygen in the lungs and brings it to cells in all parts of the body. Also called erythrocyte, RBC.
,
white blood cell
white blood cell:
Cells in the body that fight disease and infection by attacking and killing germs. There are several types of white blood cells including neutrophils, eosinophils, basophils, lymphocytes and monocytes. Each type of cell fights a different kind of germ. Also called WBC, leukocyte.
or
platelet
platelet:
The smallest type of blood cell. Platelets help the blood to clot and stop bleeding. Also called a thrombocyte.
counts.
Heparin
Brand name: Calciparine, Liquaemin
Heparin
Heparin:
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…
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.
Immunosuppressive Therapy
Immunosuppressive drug therapy
Immunosuppressive drug therapy:
(i-myoo-no-suh-PREH-siv) Drugs that lower the body's immune response and allow the bone marrow stem cells to grow and make new blood cells. ATG (antithymocyte globulin) or ALG (antilymphocyte globulin) with cyclosporine are used to treat bone marrow failure in aplastic anemia. Immunosuppressive…
lowers your body's immune response. This prevents your immune system from attacking your
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
, allowing bone marrow
stem cells
stem cells:
Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem…
to grow, which raises blood counts.
Iron Chelation
Iron chelation therapy
Iron chelation therapy:
(kee-LAY-shun) A drug therapy to remove extra iron from the body. Patients with high blood iron (ferritin) levels may receive iron chelation therapy. The U.S. Food and Drug Administratin (FDA) has approved two iron chelators to treat iron overload in the U.S. - deferasirox, an oral iron chelator,…
is the main treatment used when you have a condition called
iron overload
iron overload:
A condition that occurs when too much iron accumulates in the body. Bone marrow failure disease patients who need regular red blood cell transfusions are at risk for iron overload. Organ damage can occur if iron overload is not treated.
. 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
red blood cell:
The most numerous type of blood cell in healthy people. Red blood cells contain hemoglobin, a protein that picks up oxygen in the lungs and brings it to cells in all parts of the body. Also called erythrocyte, RBC.
transfusions.
Mycophenolate Mofetil
Brand name: CellCeptA drug that lowers the body's immune response, also referred to as an immunosuppressant drug.
Omidubicel-onlv
Brand name: OmisirgeOmisirge is the first cellular therapy approved to treat patients with severe aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… . It is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood indicated for the treatment of adults and pediatric patients 6 years or older with severe aplastic anemia following reduced intensity conditioning and for whom a compatible donor is not available.
Prednisone
Brand name: Rayos, Sterapred, Deltasone
Prednisone
Prednisone:
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.
How does it work?
For PNH, it may be used to decrease…
a man-made version of a
hormone
hormone:
A part of the endocrine system that serves as the body's chemical messengers. Hormones move through the bloodstream to transfer information and instruction from one set of cells to another.
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.
Romiplostim
Brand name: NPlate
Romiplostim
Romiplostim:
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…
is in a class of medications called thrombopoietin receptor agonists. It works by causing the cells in the
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed 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.
Tacrolimus
Brand name: Prograf
Tacrolimus
Tacrolimus:
Tacrolimus is in a class of medications called immunosuppressants. It works by decreasing the activity of the immune system. Tacrolimus can be prescribed to treat and prevent graft vs host disease (GVHD). It can also be used as part of combination therapy to treat aplastic anemia in place of…
is in a class of medications called immunosuppressants. It works by decreasing the activity of the immune system. Tacrolimus can be prescribed to treat and prevent graft vs host disease (GVHD). It can also be used as part of combination therapy to treat
aplastic anemia
aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
in place of
cyclosporine
cyclosporine:
Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure.
. Tacrolimus administration can be oral, sublingual, topical, or intravenous (IV) route.
Warfarin
Brand name: CoumadinA
blood thinner
blood thinner:
A medicine used to stop blood clots from forming. Blood thinners can be used to treat or prevent clots. Some common blood thinners are enoxaprin (Lovenox), heparin (Calciparine or Liquaemin), and warfarin (Coumadin). Also called and anticoagulant or thrombopoiesis inhibitor.
, or
anticoagulant
anticoagulant:
(ant-i-ko-AG-yuh-lunt) See blood thinner.
.