Let's talk about treatment

If you remember, PNH happens when red blood cells are destroyed by the complement system in a process called hemolysis. The two kinds of hemolysis, intravascular and extravascular, occur through two different pathways in the complement system. Prescription medications act on these pathways to help reduce hemolysis and improve symptoms, such as anemia. With the appropriate treatment, people with PNH can look forward to a near-normal life expectancy.

Prescription medications

Complement inhibitor therapy:

These medications affect the activity of specific complement proteins, namely C5 or C3, which are involved in the complement system.

• Complement C5 inhibitors: eculizumab (SOLIRIS®), ravulizumab (ULTOMIRIS®), crovalimab (PIASKY®)
• Complement C3 inhibitor: pegcetacoplan (EMPAVELI®)
• Complement factor B inhibitor (impacts C3-related activity): iptacopan (FABHALTA®)

Add-on therapy:

Factor D inhibitor, also known as danicopan (VOYDEYA™), can be used to enhance the treatment effects of the C5 inhibitors, eculizumab and ravulizumab.

Blood transfusions

Blood transfusions may be needed to replace blood cells lost through hemolysis. In this procedure, blood is normally supplied by a donor and is given through an intravenous (IV) line. Your doctor will assess your symptoms and bloodwork to determine if you need a transfusion.

Iron therapy

There can be consequences to having too little or too much iron in your body. Your doctor may prescribe iron supplements when you do not have enough. This can happen as hemolysis can lower the amount of iron in your body if it happens too quickly. Keep track of your iron! The opposite is also possible. When you receive blood transfusions or take iron supplements frequently, iron overload can happen (too much iron in your body). That is why it is important to have your iron levels monitored.

Folic acid supplements

Folic acid is a vitamin B found in fresh or lightly cooked green vegetables. It helps your bone marrow make normal blood cells. Folic acid supplements can be prescribed to help your body make blood cells.

Natural therapies

Natural therapies may include acupuncture, massage, meditation and drug-free pain management. They can help reduce pain and stress caused by the disease, or be used in combination with prescription medications to increase their effects.

Bone marrow/stem cell transplant

A bone marrow transplant involves replacing a patient's bone marrow with a healthy one from a donor. A well-matched donor can be difficult to find but is needed for the procedure to be performed. This procedure is the only cure for PNH but comes with many risks and complications. Because of this, bone marrow transplants are considered to be a last resort for patients whose PNH is very severe or who have failed all other available treatments.