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
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.
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.
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.
PNH and covid-19 research
Do PNH patients have the same resistance to Covid-19 as other people? If a PNH patient gets Covid, how does the disease progress? How does eculizumab affect this disease progression? How do PNH patients react to the corona vaccines? Here you can read the research we have found on these topics.
- COVID19 infection in patients on anticomplement therapy: The Leeds National Paroxysmal Nocturnal Haemoglobinuria service experience
- Terminal complement inhibition dampens the inflammation during COVID19
- COVID 19 Infection in A patient undergoing Treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) with Ravulizumab
- Internet based patient survey on the consequences of COVID-19 lockdown on treatment and medical follow-up of patients with aplastic anemia or paroxysmal nocturnal hemoglobinuria in Germany
- The Aplastic Anaemia Trust announces research to be undertaken by the University of Leeds and the PNH National Service on the effectiveness of COVID 19 vaccines on PNH and AA patients.