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 part of the immune system and by doing so, the medicine blocks the effect of the complement and reduces the destruction of red blood cells. It does not cure PNH 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 PNH patients). Soliris also allows patients to have the life expectancy of someone without PNH. It is also safely used in pregnancy, which previously had to be avoided by PNH patients due to the risk of blood clots.

In England and Wales, the criteria for patients being able to be treated with eculizumab can be found here.

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 in the UK 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, eculizumab is usually administered in patients’ homes by homecare nurses. For administration instructions please see here.

The homecare 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.

For more information about treatment with Ultomiris, please see the PNH National Service’s website.

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. Ravulizumab was been recommended for payment by the National Institute for Health and Care Excellence (NICE) in April 2021 for treating paroxysmal nocturnal haemoglobinuria in adults meeting the following criteria:

• with haemolysis with clinical symptoms suggesting high disease activity, or

• whose disease is clinically stable after having eculizumab for at least 6 months. 

For more details about the criteria for treatment with ravulizumab 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.

For more information about treatment with Ultomiris, please see the PNH National Service’s website.

Pegetacoplan (Aspaveli (Europe) or Empavali (USA))

Pegcetacoplan is a C3 inhibitor delivered by a sub-cutaneous injection into the abdomen twice per week. It was been licensed for use in the United States of America (by the U.S Food and Drug Administration) and in Europe (by the European Medicines Agency) and received a Great Britain Marketing Authorisation from the Medicines Healthcare Regulatory Authority (MHRA) in early 2022.

On 9 March 2022, NICE published its final guidance on pegcetacoplan on its website. NICE recommended pegcetacoplan as an option for treating adults with PNH in England and Wales who are anaemic after treatment with a C5 inhibitor for at least three months. Here are the criteria for treatment with pegcetacoplan.

Aspaveli is the trade name for pegcetacoplan in Europe and the UK and it is called Empaveli in the US.

For more information about treatment with Aspaveli please see the PNH National Service’s website.

New Drug Development

There are a number of other drugs in development to treat PNH (including biosimilars) which vary in the way they are delivered i.e. pill, daily injection or 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.

If you would like to see an overview of current PNH clinical trials taking place globally, please see here. This overview is updated three times per year.

Biosimilars

A biosimilar medicine mimics the effects of a previously approved biological medicine and is intended to treat the same disease or diseases. Eculizumab and ravulizumab are both biological medicines. Biosimilars should not be confused with traditional generic medicines, which are considered to be identical to the original medicine. There are two biosimilars for eculizumab available in the UK:

  • Bekemv – is licensed for use in adults and children aged 2 and over for PNH
  • Epysqli is licensed for adults and children for PNH

For more information on these biosimilars please see here.

Other supportive treatments for PNH 

Allogeneic Bone Marrow Transplant

It is not common for a bone marrow transplant 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 a hospital 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.