Current Treatments

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 licensed for use in Europe since 2007 and it is administered by way of intravenous infusion once every two weeks on an ongoing basis.

Soliris is a monoclonal antibody which blocks the complement part of the immune system. It is designed to attach to the C5 protein, which is part of the complement system and by doing so, the medicine blocks its effect and thereby reduces the destruction of red blood cells. It is not curative but dramatically reduces symptoms, the most significant of which is the occurrence of blood clots (which used to be the main cause of death in patients) and allows patients to have the life expectancy of someone without PNH. It has also more recently been used safely in pregnancy, which previously had to be avoided by PNH patients due to the risk of blood clots.

Although available in approximately 40 countries, unfortunately, this treatment is not available to all PNH patients globally due to its very high cost (approximately £300,000 per person per year). Bone marrow transplants can cure PNH but are rarely used due to the risk of severe complications.

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.

Ravulizumab (Ultomiris)

Ravulizumab was licensed for use in adults with PNH in the United States of America (by the U.S Food and Drug Administration) in December 2018 and in Europe (by the European Medicines Agency) in July 2019. It depends on the Health Technology Assessment process in individual countries in Europe as to whether this treatment is available to patients. Ravulizumab is currently being appraised by the National Institute for Health and Care Excellence. For more information please see here.

Like Soliris, Ravulizumab is also a monoclonal antibody which blocks the complement part of the immune system (also by attaching to C5). It is delivered by 8 weekly infusion into the vein on an ongoing basis.

To hear Dr Austin Kulasekararaj discussing Ravulizumab please watch this video (2018)

New Drug Development

There are a number of other drugs in development to treat PNH (including biosimilars) 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, please speak to your haematologist or your clinical nurse specialist at your PNH National Service Centre.

All clinical trials which are currently recruiting are listed in the Clinical Trials Register which you can access here. In the search box, type “PNH" into “Condition or disease" and the relevant country into “Country".

Hear Professor Peter Hillmen discuss a Phase III study evaluating the efficacy and safety of APL-2 (pegcetacoplan), a new C3 inhibitor here (2020)

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.

Anticoagulation

Some patients take medication to thin their blood (such as warfarin or heparin) to reduce the risk of developing blood clots.

Blood Transfusions

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

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.