PNH 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.
Antithymocyte Globulin
Brand name: ATG, Atgam, ThymoglobulinATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine 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 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 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
Blood Clot Treatment
Blood clots are very dangerous. If you have a blood clot 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 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 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 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) 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) 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 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".