PNHSource

Choose your language:

Treatment Options

What treatment options are available for PNH?1,2

There are several treatment options available for people with PNH. As you move forward, you and your healthcare team will develop a plan that’s best for you. The following approaches are most commonly used in PNH:

Supportive therapies:

Some of the supportive therapies that are currently being used to help ease the symptoms of PNH include transfusions and anticoagulants (Blood thinners)

Transfusions are recommended during times of severe haemoglobin depletion when your body cannot generate enough new cells to make up for those lost to haemolysis. It may be used on a periodic basis when your haemoglobin level is steadily decreasing.

Your physician may prescribe anticoagulants to you, in order to either reduce the risk of getting blood clots or may need to dissolve a clot when it happens.

Complement Inhibition:

When unregulated, the complement can lead to various severe diseases causing damages in multiple organs. Complement inhibitors are compounds which bind to enzymes in the complement system. Their role is to suppress or reduce the activity of complement.

Bone Marrow Transplantation:

Bone marrow transplantation (BMT) is the only known treatment that may cure PNH. It involves replacing the body’s defective blood stem cells by new healthy ones from a donor. However, bone marrow transplantations carry significant risks which should be discussed with your doctor.

It is important to remember that symptoms of PNH may vary from patient to patient, and a plan to manage your PNH may work for you but may not work for someone else. This is why it is so important to discuss treatment options with your doctor.

Living with PNH

References: 1. Brodsky RA. Paroxysmal nocturnal haemoglobinuria. In: Hoffman R, Benz EJ Jr, Shattil SJ, et al, eds. Hematology: Basic Principles and Practice. 4th ed. Philadelphia, PA: Elsevier Churchill Livingstone; 2005:419-427. 2. Rosse WF. Treatment of paroxysmal  nocturnal haemoglobinuria. Blood. 1982;60:20-23.