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How is PNH treated?

Medical teams may use various treatments for PNH depending on the individual patient’s signs, symptoms and blood results. PNH patients should discuss the possible treatment options with their medical team and agree the best approach for their individual case and circumstances.

Blood transfusions

Blood taken from donors can be given to PNH patients to increase their levels of red blood cells and improve the symptoms of anaemia they experience. Blood transfusions may need to be repeated at regular intervals.

Vitamin and mineral supplements

Vitamin and mineral supplements such as iron and folic acid may be given, as the body needs them to produce new red blood cells.

Anticoagulants

Anticoagulants are drugs that thin the blood to try to stop it from forming blood clots. Warfarin is an example of an anticoagulant medicine.

Hormones

Hormones such as steroids and androgens may be given to try to stop the destruction of red blood cells.

Drugs that suppress the immune system

Some drugs that decrease the effect of the immune system may be given to treat PNH to try to stop the destruction of red blood cells. Other drugs which decrease the effect of the immune system may also be given to treat bone marrow problems, if they are also present.

Bone marrow transplantation (BMT)

BMT is a process of removing a person’s own bone marrow cells and replacing them with a donor’s cells. It can remove the cells that produce PNH blood cells. It carries quite high risks and needs the PNH patient to have a suitable matching donor, which is often not the case. Because of this, bone marrow transplantation is often only used in PNH when there is also a serious problem with bone marrow function.

It is important to remember that some PNH patients will not require any treatment but just have their PNH monitored over time in case the situation changes.

In a small number of patients PNH can actually disappear on its own, which is called spontaneous remission. This is another reason why it is important for the disease to be carefully monitored to see if it changes.

Complement inhibitor therapy

Complement inhibitor therapy blocks complement (part of your body's defence system). By doing so, the red blood cells that are missing protective proteins (PNH blood cells) are protected from destruction.