Paroxysmal Nocturnal Hemoglobinuria (PNH)
Amy E. DeZern, MD, MHS
Michael Keng, MD
Rami Komrokji, MD
Stephanie Halene, MD, PhD
Patient Advisory Council
The AAMDSIF Patient Advisory Council (PAC) was created in 2008. Its purpose is to serve as a resource to the AAMDSIF staff in the development and review of patient education materials, programs and services.
Special Issues for People with PNH
Because you have PNH, everyday events can be more risky for you than for healthy people. Here are some examples.
How do I get my medical records from a doctor?
If you want a copy of your medical records, you must submit a written request to the hospital or the doctor's office. The law requires that patients provide doctors with written authorization to disclose medical records or the information contained in them. Some hospitals have their own form that needs to be completed. Patients may receive copies of their medical records, however the originals must remain in the physician's or hospital's file. Please consult with your physician's office for access to digital records.
How long can I stay on cyclosporine?
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.
What are the side effects of cyclosporine?
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.
