Eculizumab (Soliris) is used to treat patients with PNH who need therapy (not all patients do) and who meet the national criteria for treatment. It was approved for treatment in England in 2007. Eculizumab is an antibody that blocks the part of the immune system called complement, that is responsible for attacking the blood cells in PNH. By blocking complement it reduces or stops the destruction of the PNH blood cells as well as preventing other complications due to unregulated complement activity. Eculizumab has been shown to reduce or stop many of the symptoms of the disease and thereby improve the quality of life of people treated. It also reduces the risks of many of the complications of PNH, such as thrombosis, renal failure or pulmonary hypertension (high blood pressure in the lungs). It has also therefore improved life expectancy in certain groups of patients with PNH.
What are the risks?
Patients on eculizumab can still clear the majority of infections with other parts of their immune system. However, patients are at a slightly higher risk of getting infections from one particular strain of bacteria, the Neisseria species. The infection we worry about the most is therefore that which may result in meningitis. This is because eculizumab blocks the part of the immune system that usually protects us from these particular bacteria. This risk is still extremely low but to reduce this even further we vaccinate patients who are to receive this drug against this bacterium. The vaccine does not currently cover all the strains of the bacterium and to further reduce the risk of getting an infection we additionally advise patients to take daily preventative (prophylactic) antibiotics.
In patients who feel unwell or develop a fever while receiving eculizumab it is important to receive urgent medical attention. Contact numbers will be provided, including a 24 hour medical emergency number for the PNH Service in Leeds. Patients should also receive a local emergency contact number. Patients who are unwell are highly likely to need to attend the local department where observations will be taken and blood tests, including blood cultures to look for bacteria in the blood stream.
Side effects
Most patients tolerate eculizumab extremely well and suffer from no symptoms due to the treatment. The most commonly reported adverse reaction is a headache which is quite common after the first or second dose of eculizumab but usually is not a problem with subsequent doses. Dizziness, nausea and a temperature are very occasionally reported each occurring in around 1 in 20 (5%) of people.
How is eculizumab given?
Eculizumab is given into the bloodstream (intravenous), through a small needle in the hand. It takes approximately 30 minutes to administer and will be given by a nurse. The first two to five infusions are usually given in hospital and subsequent ones will be in your own home with the nurse visiting you (see Homecare service).
The treatment is given once a week at a dose of 600mg for the first 4 weeks then 900mg every 2 weeks from the fifth week onwards. A few patients require a higher dose of eculizumab and this will be monitored and increased by the PNH team.
It is important that the treatment is given every 14 days and no doses are missed. However there is some flexibility, (up to 2 days) but this must be agreed with the PNH Centre.
If you are booking a holiday please call the PNH team to discuss your holiday dates giving as much warning as possible (at least 2 months is ideal if we need to modify your treatment around the holiday). If you are looking at travelling for longer than 2 weeks it may be possible to arrange treatment in another location in the UK or abroad but this usually requires at least 2 months to sort out the logistics (see Travel information).
Eculizumab Homecare service
For patients receiving treatment with eculizumab in the UK there is a homecare service enabling treatment to be given at home rather than in hospital. The homecare team work as an extension of the PNH team. Patients receive up to 5 infusions (usually 2) in hospital before being transferred to the homecare service.
The transfer to homecare will be discussed during one of the visits to see the PNH team. The Homecare customer service team will then telephone to make arrangements for the first infusion at home. A nurse will administer the treatment. They will make contact at least 24 hours before the next infusion is due to be administered to arrange a convenient time. The nurses work closely with the PNH centre.