There are different treatments that those living with PNH may require. Some people, especially those with a small PNH clone may require little or no treatment.
Eculizumab (or Soliris)
“Soliris” has been approved for use in the UK since 2007 and many PNH patients receive it and it has transformed the quality of life and life expectancy of many patients. Your haematologist will be able to tell you if you fulfil the criteria to be treated with this drug.
Eculizumab is a monoclonal antibody which blocks the activation of the complement part of the immune system. It therefore protects the red blood cells which are affected by PNH from being destroyed by the complement part of the immune system due to them having the usual protective covering.
This drug is administered by way of intravenous infusion once every two weeks on an ongoing basis.
Eculizumab has been proven to reduce the symptoms of PNH patients and improve their quality of life together with their life expectancy. It also reduces the risks of many of the complications of PNH such as thrombosis, renal failure or pulmonary hypotension.
Terminal complement (which eculizumab prevents from forming) is required to prevent Neisseria Meningitidis which is a bacteria which can cause meningitis and other forms of meningococcal disease. Therefore patients treated with eculizumab are more likely to develop infections caused by the Neisseria group of bacteria. To reduce this risk, all PNH patients starting treatment with eculizumab are vaccinated with a vaccine against the meningococcal strains A, C, W and Y. The vaccine against serotype B (the most common serotype in the UK) is licensed and is used in patients treated with eculizumab. Patients are also advised to take 500mg of either penicillin or erythromycin twice daily as a prophylactic antibiotic to protect them from infection.
Patients treated with eculizumab are provided with a patient safety card to carry with them at all times which states the symptoms of meningitis and tells them what to do if they experience any of these symptoms.
In the UK, eculizimab is usually administered at patients’ homes by homecare nurses.
The nurse attends a patient’s home with the medication (or it is delivered separately and kept in the fridge) and administers an intravenous line into a vein and flushes the line with saline. The prescribed amount of eculizumab is then mixed with saline according to the prescription and this is then usually administered over a 30 minute period. After the eculizumab has been infused, the line is then flushed with saline again. Blood pressure and temperature are taken before and after the infusion in case there is any change which may indicate an adverse reaction.
New Drug Development
There are a number of other drugs in development to treat PNH (including biosimilars –insert link to this section in Research) and they vary in the way they are delivered i.e. pill, daily injection, 8 weekly infusion. If you are interested in taking part in a clinical trial (insert link to research page) please speak to your haematologist or your clinical nurse specialist at your PNH National Service Centre.
Other treatments for PNH are listed below:
Allogeneic Bone Marrow Transplant
It is not common for this treatment to be recommended for PNH patients unless there is a severe bone marrow failure co-existing with PNH. It has many significant complications which may occur in some patients.
Some patients take medication to thin their blood (such as warfarin or heparin) to reduce the risk of developing blood clots.
Blood transfusions can alleviate some of the symptoms experienced with PNH, including anaemia. A blood transfusion can usually be carried out in an outpatient clinic. Once your blood type is cross-matched for antibodies, one unit of blood takes approximately two hours to transfuse into your body. This will usually alleviate the symptoms resulting from anaemia and haemolysis for a period of time (which differs for each person).
Folic acid is a vitamin which the bone marrow needs to help it produce blood cells. This can be taken in tablet form.
Iron Supplements and Iron Removal
Iron levels can be both too high or too low in PNH and can depend on what other treatment someone is having. Some patients may need to take iron supplements such as ferrous sulphate and others may need to take medication to reduce the iron in their bodies.