There are a number of different treatments that patients with PNH may require. Some people, especially those with a lower proportion of PNH cells may require little or no treatment. Below is a list of some of the more common treatments in use.
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 symptoms 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.
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.
Ravulizumab
Ravulizumab (or Ultomiris) is an intravenous infusion that is administered every 8 weeks in the long-term. The first 2 doses are given 2 weeks apart.
Ravulizumab blocks the activation of complement and therefore protects the PNH cells from destruction or stimulation. Ravulizumab works in the same way as Eculizumab, the structure of the drug has been changed to create a longer lasting treatment. As soon as Ravulizumab 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 Ravulizumab is a treatment that needs to be given lifelong.
Ravulizumab 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.
Ravulizumab lasts longer so is therefore the preferred treatment option for patients outside of a clinical trial who require complement inhibitor. PNH patients who were on Eculizumab have now been offered to switch to Ravulizumab if suitable and this rollout process is ongoing.
Clinical trials
Treatment for PNH has progressed significantly in the last 20 years. We are now in a position to look at optimising treatment for patients in terms of modality of treatment and managing anaemia for patients experiencing extravascular haemolysis.
The National PNH service has clinical trials currently open (Leeds, Kings and Monklands) for patients who are new to treatment and those already on treatment with eculizumab or ravulizumab. These include self injection infusions or short injections, oral treatments or combination of intravenous or oral treatments.
We aim to offer all patients with PNH who are eligible a clinical trial option for them to consider, and this will be discussed at clinic appointments
Prevention of meningococcal infection: vaccination and antibiotics
Individuals treated with complement inhibition (eculizumab, ravulizumab or within a clinical trial) 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.