PNH is considered a chronic disease meaning that it lasts a long time. The only potential cure is a bone marrow transplant (BMT). 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: Consist of therapies to help manage the symptoms of your PNH. They work to increase blood counts. Treatment is typically blood transfusions and may include growth factors 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: Lowers your body's immune response and is appropriate for PNH patients who also have aplastic anemia. This therapy uses medicines to keep the immune system from attacking the bone marrow. Antithymocyte globulin (ATG) and cyclosporine are the medicines typically used.
- Eculizumab (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 less active and reduces hemolysis Soliris ® is approved for the treatment of patients with PNH in nearly 50 countries worldwide.
- 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.
- Bone marrow/stem cell transplantation (BMT/SCT): A procedure that replace your unhealthy blood-forming stem cells 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: Also called research studies, they may be an option for patients who do not have success with other treatment options.