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Page last updated: 3rd October 2024
Leukaemia Care and UK Masto successfully campaign for access to a new treatment for patients with mastocytosis in England and Wales
Leukaemia Care and UK Masto are pleased to announce that Avapritinib (Ayvakyt©) has been approved for treating advanced systemic mastocytosis (including aggressive systemic mastocytosis, systemic mastocytosis with an associated haematological neoplasm, and mast cell leukaemia) in adults in England and Wales, thanks to a decision made by the National Institute for Health and Care Excellence (NICE) today.
Today (Thursday 3rd Otober 2024), Leukaemia Care and UK Masto welcome the decision by the National Institute for Health and Care Excellence (NICE) to approve Avapritinib (Ayvakyt©) for use on the NHS in England and Wales. This is a treatment for adult patients with advanced systematic mastocystosis, (including aggressive systemic mastocytosis, systemic mastocytosis with an associated haematological neoplasm, and mast cell leukaemia).
Your own treatment options may also depend on your suitability for treatment when you need it, such as whether your platelets are high enough. Please check with your healthcare team or speak to Leukaemia Care or UK Masto if you have questions about your eligibility for treatment.
Northern Ireland will likely follow NICE’s decision. However, avapritinib is not currently available on the NHS in Scotland. We will update this article once we have further updates on the availability of avapritinib in Northern Ireland and Scotland.
Leukaemia Care alongside charity UK Masto submitted a joint written response to NICE representing the views of patients. A submission was created by drawing on experiences of individual patients and data from our surveys.
Jess Hobart, from UK Masto, said:
“We welcome the decision by NICE to approve this treatment and are pleased that both parties were able to reach an agreement without further delay in access for patients. The approval of avapritinib, which has been shown to perform better than existing treatments, is important for clinicians to tailor treatment plans to their patients effectively.”
Leukaemia Care’s CEO, Colin Dyer, said of today’s decision:
“Patients with advanced systematic mastocytosis currently have a limited number of treatment options available to them. This includes those with mast cell leukaemia, where survival rates are only around 6 months after diagnosis. A new option for these patients to have improved survival and better quality of life after such a serious diagnosis is very welcome.”
Mastocytosis is a type of mast cell disease. It is a rare condition caused by the build up of mast cells in the body’s tissue. There is no cure for mastocytosis, so the aim of treatment is to try to relieve the symptoms. Treatment options will depend on the type of mastocytosis and the severity of the symptoms.
There are two main types; indolent (slow growing) and advanced (develops faster and is more immediately life threatening). The treatment approved today is for advanced systemic mastocytosis only. Some of the symptoms of advanced mastocytosis can include anaphylaxis, cramping and diarrhoea, upper gastrointestinal pain due to hyperacidity, skin rash/hiving/itching, bone pain, and vomiting.
Mast cell leukaemia is the most aggressive form of advanced systemic mastocytosis, although it is extremely rare. Currently, the life expectancy of someone diagnosed with mast cell leukaemia is only 6 months on average.
Before the approval of avapritinib, patients would usually be treated with midostaurin. While patients we spoke to were grateful that it is available and funded, it is not curative, some patients found that it stopped working after some time, and it causes significant gastrointestinal symptoms in many patients (vomiting is very common). Patients with advanced mastocytosis need medications that are easily tolerated and more effective at preventing symptoms and prolonging life.
Evidence from avapritinib trials (Pathfinder and Explorer), as well as real world data from patients in the UK receiving it via compassionate use, shows that avapritinib has better outcomes than the other medications currently available in the UK. Patients also reported that this new treatment reduced the severity of their symptoms and had less intense side effects than midostaurin.
Avapritinib is an oral treatment that is taken in the form of a tablet. It is likely that it can only be prescribed by a haematologist. Please contact either UK Masto or Leukaemia Care if you have any concerns or questions about accessing this treatment.
Support services
If you or a loved one needs advice or support regarding your diagnosis, please get in touch. You can call our free helpline on 08088 010 444 and speak to one of our nurses. Alternatively, you can send a message to our team via WhatsApp on 07500 068 065 (services available Monday to Friday, 9am – 5pm).