What are the treatments for PNH?

PNH is considered chronic. That means it lasts for a long time. The only known cure is a bone marrow transplant. Other treatments are designed to ease symptoms and prevent problems.

There are several treatments and treatment approaches your doctor may consider. These include the following:

• Wait and watch, also called “watchful waiting,” is an approach your doctor might suggest if your blood counts aren't too low and you symptoms aren't too bad.
• Supportive care helps you manage the symptoms of your PNH. Its primary goal in PNH patients is to increase blood counts. Supportive care treatments typically include blood transfusions, and may include growth factors. For PNH patients this might also include taking extra iron (iron therapy).
• Blood thinners (anticoagulants) may be used on some patients to help reduce the chance of getting blood clots.
• Immunosuppressive therapy can lower your body's immune response and is appropriate for PNH patients who may 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®) is the only 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 (a part of your immune system) less active.

• Bone marrow/stem cell transplantation replaces your unhealthy blood-forming stem cells with healthy ones.

Clinical trials, also called research studies, may also be an option for patients who do not have success with the other treatment options.