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Hydroxyurea
Hydroxyurea:
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.
is in a class of medications called antimetabolites. Hydroxyurea treats cancer by slowing or stopping the growth of…
Drug last updated
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It is used to prevent blood clots from forming.
Warfarin
Warfarin:
It is used to prevent blood clots from forming. Warfarin is a tablet that is taken by mouth.
is a tablet that is taken by mouth.
Drug last updated
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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…
Drug last updated
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Pozelimab
Pozelimab:
Pozelimab is a monoclonal antibody inhibiting the pathway associated with haemolysis of red blood cells. The drug is intended to decrease LDH levels and the incidence of breakthrough haemolysis.
is a
monoclonal antibody
monoclonal antibody:
A type of protein called an antibody that is engineered to look for a specific substance in the body. There are many…
Drug last updated
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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…
Drug last updated
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Enoxaparin
Enoxaparin:
Enoxaparin is in a class of medications called low molecular weight heparins. It works by stopping the formation of substances that cause clots. It is also used in combination with warfarin to treat blood clots in the leg.
is in a class of medications called low molecular weight heparins. It works by stopping the formation of substances that cause…
Drug last updated
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Cytarabine (ara-C)
Cytarabine (ara-C):
Cytarabine (ara-C) is a chemotherapy drug. This medicine reduces the growth of cancer cells and can suppress the immune system. It is used for treating leukemias or lymphomas. It is often given with other cancer drugs.
is a
chemotherapy
chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People…
Drug last updated
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Cemdisiran (ALN-CC5)
Cemdisiran (ALN-CC5):
Cemdisiran (ALN-CC5) is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for the treatment of complement-mediated diseases.
is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for…
Drug last updated
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Ravulizumab-cwvz
Ravulizumab-cwvz:
Ravulizumab-cwvz (Ultomiris®) is a drug approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat PNH. ULTOMIRIS® is a long-acting C5 inhibitor that works by inhibiting the C5 protein in the terminal complement cascade. ULTOMIRIS® is a prescription medicine called a…
(Ultomiris®) is a drug approved by the U.S. Food…
Drug last updated
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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…
Drug last updated
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Daunorubicin
Daunorubicin:
Daunorubicin is in a class of medications called anthracyclines. It works by slowing or stopping the growth of cancer cells in your body. It is used in combination with other chemotherapy drugs to treat a certain type of acute myeloid leukemia (AML).
is in a class of medications called anthracyclines. It works by slowing or stopping the growth…
Drug last updated
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Folate
Folate:
(FOE-late) A B-vitamin that is found in fresh or lightly cooked green vegetables. It helps the bone marrow make normal blood cells. Most people get enough folate in their diet. Doctors may have people with paroxysmal nocturnal hemaglobinuria (PNH) take a man-made form of folate called folic acid.
is a B-vitamin that is found in fresh or lightly cooked green vegetables.…
Treatment last updated
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Clinical research
Clinical research:
A type of research that involves individual persons or a group of people. There are three types of clinical research. Patient-oriented research includes clinical trials which test how a drug, medical device, or treatment approach works in people. Epidemiology or behavioral studies look at the…
is at the heart of all medical advances, identifying new…
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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…
Treatment last updated
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Androgens
Androgens:
Androgens are natural male hormones that can cause your bone marrow to make more red blood cells. This can improve anemia. Androgens are sometimes used to treat aplastic anemia and PNH.
Androgens that may be used include:
Danazol (Danacrine®)
Fluoxymestrone (Halotestin®)
Oxymetholone
…
are natural male hormones that can cause your
bone marrow
bone…
Treatment last updated
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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…
Treatment last updated
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If you have MDS and have a high-risk or intermediate-2 risk IPSS score, you have a higher risk of developing
Acute Myelogenous Leukemia
Acute Myelogenous Leukemia:
(uh-KYOOT my-uh-LAH-juh-nuss loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous…
Treatment last updated
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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…
Treatment last updated
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Diseases like
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…
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myelodysplastic syndromes…
Basic page last updated
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The most common white blood cells are called neutrophils. They fight infection. Patients who don't have enough healthy neutrophils are said to have
neutropenia
neutropenia:
(noo-truh-PEE-nee-uh) A condition in which there is a shortage of neutrophils in the bloodstream. Neutrophils are a type of white blood cell. This results in a low white blood cell count.
. Because of their low white…
Treatment last updated
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