Paroxysmal Nocturnal Hemoglobinuria (PNH)

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Cyclosporine Cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. is an immunosuppressive drug that works by preventing T-lymphocytes from becoming active. Once the T-lymphocytes are turned off, they stop attacking 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… in the bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. . That means stem cells may be able to grow back. It is used along with antithymocyte globulin antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. (ATG) as a standard treatment for 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… , and may also be used to treat MDS and PNH.

Some aplastic anemia patients may require prolonged ongoing treatment with cyclosporine to prevent or minimize the risk of disease relapse aplastic anemia coming back. Generally speaking, patients can remain on cyclosporine indefinitely, as long as there is evidence that the drug is providing some benefit and there are no unwanted or adverse side effects.

For patients who respond well to cyclosporine, alone or in combination with antithymocyte globulin (ATG), hematologists may reduce the cyclosporine dosage. Tapering, or reducing, the dosage is done slowly over a long period of time. If low blood counts and disease symptoms return, your doctor may increase or restart cyclosporine. For those who experience bad side effects such as kidney toxicity, the drug must be discontinued.

Cyclosporine Cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. is a drug that lowers your body's immune response. Cyclosporine is used along with antithymocyte globulin antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. (ATG), another immunosuppressant, for treating 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… and some other 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. If you are taking cyclosporine as part of your treatment regimen, your doctor should monitor you for side effects. Cyclosporine may cause high blood pressure, kidney damage, loss of magnesium or potassium, liver inflammation, increased hair growth and gum swelling.

These problems can usually be fixed. Your doctor might prescribe medicine to lower your blood pressure and to replace magnesium or potassium.

It is important to weigh both the risks and the benefits of a bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. /stem cell transplant. A transplant doctor can answer your questions and help you decide if a transplant is an option for you.

A bone marrow transplant bone marrow transplant: A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due… has serious risks. Some patients suffer from life-threatening problems as a result of their transplant. These problems can include serious infections and graft-versus-host disease (GVHD) graft-versus-host disease (GVHD): Also called GVHD, it is a common complication of bone marrow/stem cell transplantation. It is caused when the donor's immune cells, now in the patient, begin to see the the patient's body as foreign and mount an immune response. GVHD most commonly effects the recipient's skin, intestines, or liver… , in which the transplanted cells attack the patient's body.

For some patients, a transplant may be the best option for a longer life, as the procedure remains the only known cure for 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 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 and PNH. You, your doctor and your family need to consider many factors when making a decision on a transplant, including your disease stage, your age, your overall health, whether a matching donor is available and other treatment options.

Doctors will sometimes refer to certain treatments as supportive therapy or  supportive care supportive care: Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive… . They help you manage the symptoms of your disease, although they do not treat the underlying cause of the disease.

In general, supportive care includes the following:

• Blood transfusions Blood transfusions: A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, 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… to raise blood cell counts
• Antibiotics 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. Because of their low white count, they may get infections easily and have trouble getting rid of infections. If you have neutropenia,… to treat infection
• 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,… to treat 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. , a side effect of having many 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
• 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… that are man-made versions of natural hormones that tell your body to make more blood cells.