Eculizumab

Eculizumab (or Soliris) is an intravenous infusion that is administered every 2 weeks in the long-term. Eculizumab blocks the activation of complement and therefore protects the PNH cells from destruction or stimulation. As soon as eculizumab therapy is stopped, complement will become active and the PNH cells that were previously protected will be vulnerable to complement attack again. It is therefore usual that eculizumab is a treatment that needs to be given lifelong.

Eculizumab has been proven to reduce the symptoms experienced in PNH and has been shown to improve people’s quality of life. It also reduces the risks of many of the complications of PNH, such as thrombosis, renal failure or pulmonary hypertension (high blood pressure in the lungs). It has also therefore improved life expectancy in certain groups of patients with PNH.

Blood Transfusions

Anaemia is one of the commonest symptoms of PNH. Blood transfusions are an important therapy as they can alleviate some of the symptoms experienced. We generally only use transfusions if the patient has symp-
toms of anaemia.

Folic Acid

Folic acid is a vitamin needed by the bone marrow to help make blood cells. In PNH, the bone marrow often produces more red blood cells than normal to try to compensate for the blood cells being destroyed in the bloodstream. Taking folic acid tablets helps to ensure that the bone marrow has enough folic acid to make blood cells.

Anticoagulation

Some patients are on blood thinning medication, such as warfarin, to reduce the risk of developing blood clots. These medications make people more likely to bruise and bleed.

Iron supplements and iron removal

Iron levels may be either too low or too high in PNH, usually depending on whether the patient is on specific treatment for the PNH or not. Some patients therefore may require iron supplements and others may need medication to reduce the iron levels in the body.

Prevention of meningococcal infection: vaccination and antibiotics

Individuals treated with eculizumab are at increased risk of infection by a specific bacterium (Neisseria Meningitidis). Both vaccination against Neisseria Meningitidis and preventative antibiotics are used to reduce this potential risk.

Allogeneic Bone Marrow Transplantation

Bone marrow transplant is the only curative treatment for PNH but has many significant complications which may occur in some patients. It is not very common for this to be recommended for patients with PNH unless there is severe bone marrow failure co-existing with PNH.