PNH is considered a chronic disease meaning that it lasts a long time. The only potential cure is 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. . However, a BMT carries many risks and is not an option for many people. Other treatments are designed to ease symptoms and prevent problems. These may include:
- Wait and watch: Also called “watchful waiting,” your doctor might decide to do nothing but monitor your blood counts if they aren't too low and your symptoms aren't too bad.
- 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… : Consist of therapies to help manage the symptoms of your PNH. They work to increase blood counts. Treatment is typically 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… and may include 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… or taking extra iron (iron therapy).
- Blood thinners (anticoagulants) may be used on some patients to help reduce the chance of having blood clots.
- 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… : Lowers your body's immune response and is appropriate for PNH patients who also have 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… . This therapy uses medicines to keep the immune system from attacking the bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. . 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) and cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. are the medicines typically used.
- Eculizumab Eculizumab: Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes. You will probably get an IV once a week for the first 4 weeks. Starting in the 5th week, you will get a slightly higher dose of Soliris every 2 weeks. … (Soliris ®) was the first drug approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Evaluation Agency (EMEA) to treat PNH. It works by making your complement system complement system: A group of proteins that move freely in the bloodstream. These proteins support (complement) the work of white blood cells by fighting infections. less active and reduces hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. Soliris ® is approved for the treatment of patients with PNH in nearly 50 countries worldwide.
- 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 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.
- Bone marrow/stem cell transplantation (BMT/SCT): A procedure that replace your 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 from a matched donor. BMT is the only potential cure for PNH. Unfortunately, BMT is an “imperfect cure,” carrying many risks and potential long-term side effects. For many people a BMT is not a good option. When considering BMT, be sure to talk with your doctor about its potential impact on your long-term survival and quality of life when compared with other treatment options.
- Clinical trials 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… : Also called research studies, they may be an option for patients who do not have success with other treatment options.