PNH National Services

Ravulizumab Treatment

Ravulizumab (Ultomiris) is used to treat patients with PNH who need therapy (not all patients do) and who meet the national criteria for treatment.

Ravulizumab was approved for treatment in the UK in 2020. It 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.

Benefits of treatment:

  • Improved life expectancy in certain groups of patients with PNH.
  • Reduce or stop many of the symptoms of the disease and thereby improve the quality of life of people treated.
  • Reduces the risks of many of the complications of PNH, such as thrombosis, renal failure or pulmonary hypertension (high blood pressure in the lungs).

What are the side effects?

Patients on Ravulizumab can still clear the majority of infections with other parts of their immune system.

Patients are at a slightly higher risk of getting infections from one particular strain of bacteria, the Neisseria species, which may result in meningitis. This is because Ravulizumab blocks the part of the immune system that usually protects us from these particular bacteria.

If patients on complement inhibition are unwell:

In patients who feel unwell or develop a fever, 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 or London. 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.

How is Ravulizumab given?

Ravulizumab is given into the bloodstream (intravenous), through a small needle in the hand. It takes approximately 45 minutes to administer and will be given by a nurse. The first infusion is usually given in hospital and subsequent ones will be in your own home with the nurse visiting you (see Home care service).

The treatment is given, depending on weight, on day 1, day 15 then every 8 weeks after this. It is important that the treatment is given every 8 weeks and no doses are missed. However, there is some flexibility, (up to 7 days) but this must be agreed with the PNH Centre.

If you are booking a holiday, where possible please avoid booking at the time of your treatment. If this is not possible, 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 – see Travel information).

Ravulizumab Home Care Service

For patients receiving treatment with Ravulizumab in the UK, there are two home care services – Sciensus and Alcura – enabling treatment to be given at home rather than in hospital. The home care teams work as an extension of the PNH team.

The transfer to home care will be discussed during one of the visits to see the PNH team. The home care 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.