treatments;
standard therapy

1. 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 and are required to be vaccinated against these. In some countries, patients are also advised to take daily prophylactic antibiotics 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.

2. Ravulizumab (or Ultomiris®)

Ravulizumab was licensed for use in adults with PNH in the Unites 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. 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.

3. New Drug Development

Biosimilars of eculizumab are in the making. They hopefully help the price to go down by competition. More advanced medicines are in the pipeline who inhibit not only C5, but also C3. As a C5-inhibitor checks the hemolysis in the blood vessels, a C3-inhibitor does just that and impedes the blood breakdown in the spleen and liver also, resulting in a higher hemoglobin level. 

These new medicines are delivered  each in their own way, like infusion every fortnight or every 8 weeks, (self-administered) subcutaneous injection twice a week, a pill twice a day etc. 

For clinical trials, see hereunder. For more up to date information contact us. 

Other supportive treatments

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 hemolysis for a period of time (which differs for each person).

Anti-
coagulation

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

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.

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.

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

Clinical trials

Three times a year we update an overview of clinical trials on PNH taking place in your country. Please click here. For the latest information you may want to go to the source of our overview. You can find it here.

Wellbeing

Wellbeing encompasses many strands including diet, sleep, exercise and management of stress levels. A number of PNH patients report that stress makes their symptoms worse.

One of our members, the Canadian Association of PNH Patients has created “The Guide to Living Well” which has useful information about a number of different aspects of living with PNH including nutrition and exercise and the mental and emotional impact.

Play Video

quality of life

The PNH Global Alliance is committed to ensuring that the quality of life of patients is measured as accurately as possible in trials and other studies. It is not self-evident that higher blood values lead to less fatigue and a higher ability to concentrate; this must be demonstrated with a sophisticated measuring instrument, such as the QLQ-AA / PNH.

Patient representative Pascale Burmester from Germany has been interviewed by Dr Jens Panse about the meaning of the AA and PNH specific Quality of Life Questionnaire. Watch the video for more information! Subtitles are available in different languages. Please select a language in the settings of the video.

There are some Quality of life questionnaire for patients with AA and/or PNH. We would like to know how you have been feeling recently. Please try to answer as many of the questions as possible by ticking one of the four boxes. Download the document with questions here.

There is also a report created: Design and development of a diseasespecific quality of life tool for PNH patients. Read the document here

Here is an example of the QLQ being used in Japan.

Research

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of hematopoietic stem cells, characterized by hemolytic anemia, bone marrow failure, and thrombosis. A multinational phase III study reported that eculizumab, C5 monoclonal antibody therapy, effectively prevents complement attacks in patients with PNH who suffer from severe hemolytic attacks. As a result, both the levels of complement-induced hemolysis and quality of life (QOL) in patients with high disease activity have improved dramatically.