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.

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Alemtuzumab

Brand name: Campath
A monocolonal antibody - a medicine that is engineered to look for a specific substance in the body. Alemtuzumab: Alemtuzumab is a drug used in the treatment of chronic lymphocytic leukemia (CLL), cutaneous T-cell lymphoma (CTCL) and T-cell lymphoma under the trade names Campath, MabCampath and Campath-1H, and in the treatment of multiple sclerosis as Lemtrada. It is also used in some conditioning regimens for… attaches to and kills white blood cells called lymphocytes. In certain types of 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… , lymphocytes are responsible for attacking the bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. 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… . Alemtuzumab is in clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… 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 treating other conditions that require immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… .

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: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. to make more red blood cells. This can improve anemia: (uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness. . Androgens are sometimes used to treat 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… and PNH.

Antibiotics

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: (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 count, they may get infections easily and have trouble getting rid of infections.
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Antithymocyte Globulin

Brand name: ATG, Atgam, Thymoglobulin

ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. as the first-line immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… (IS) to treat patients with acquired 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 some cases it is used as a treatment for patients with MDS and PNH.

Biosimilars

A biosimilar is a type of biologic medication. The term “biosimilar” means that the medication is highly similar to the already approved brand name biologic (“originator” medication). Biosimilars: What is a Biosimilar? A biosimilar is a type of biologic medication. The term “biosimilar” means that the medication is highly similar to the already approved brand name biologic (“originator” medication). Biosimilars are not generic versions of the original biologic. Difference Between… are not generic versions of the original biologic.

Blood Clot Treatment

Blood clots are very dangerous. If you have a blood clot: A mass of blood that forms when platelets stick together. Harmful blood clots are more likely to happen in PNH. The term thrombus describes a blood clot that develops and attaches to a blood vessel. The term embolus describes a blood clot or other foreign matter that gets into the bloodstream and… , you need to get help right away. Patients with PNH are at an increased risk of getting serious blood clots. Here are some ways blood clots are treated.

Blood Transfusions

A blood transfusion: A procedure in which whole blood or one of its components is given to a person through an intravenous (IV) line into the bloodstream. A red blood cell transfusion or a platelet transfuson can help some patients with low blood counts. is a safe and common procedure. Most people who have a 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… disease like 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… , MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some patients with low blood counts.

Bone Marrow Transplant

A bone marrow transplant (BMT): A procedure where bone marrow stem cells are collected from marrow inside the donor's hipbone and given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells. is also called a stem cell transplant (SCT): A procedure where blood-forming (hemapoietic) stem cells are taken from a healthy donor. They enter the patient's bloodstream through an intravenous (IV) needle and make their way into the bone marrow. The donor cells are called a graft. There are three sources of stem cells - bone marrow, cord… or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming 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… with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due to the risks and potential long-term side effects as an "imperfect cure".

Clinical Trials

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 ways to prevent, detect or treat disease. If you have a 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… disease, you may want to consider taking part in a clinical trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a… , also called a research study.
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Cyclophosphamide

Brand name: Cytoxan
Cyclophosphamide: Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system. is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat 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… , it works by suppressing your body's immune system.

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