Liposomal cytarabine with daunorubicin approved for use in Scotland for t-AML and AML-MRC

The Scottish Medicines Consortium (SMC) has today recommended the use of liposomal cytarabine with daunorubicin to treat previously untreated therapy-related AML (t-AML) and AML with myelodysplastic changes (AML-MRC).

This follows the recent news that the National Institute for Health and Care Excellence (NICE) have also recommended that this drug be used on the NHS in England. The brand name for this treatment is Vyxeos and it is manufactured by Jazz pharmaceuticals.

Liposomal cytarabine is a chemotherapy drug which works by imitating a part of DNA called cytidine. Cells replace the cytidine with the drug cytarabine in their DNA. The presence of cytarabine in place of cytidine then stops the cell from being able to replicate the DNA and causes the cell to die. Cytarabine is already used to treat acute myeloid leukaemia (AML), but Vyxeos is different in that the cytarabine is encased in droplets of fat molecules called liposomes. It is thought that doing this may make the cytarabine work better and last longer in the body, hence why it is has been proposed as an alternative treatment to standard cytarabine.

Both kinds of cytarabine are delivered with daunorubicin, which also works by preventing DNA from replicating, but it does this in a different way to cytarabine. It is called an intercalator, meaning it sits in gaps in the DNA, stopping enzymes in the cell from copying or fixing the DNA. Again, this leads to the death of the cells. It is targeted at the leukaemia cells by being delivered into the bloodstream. AML patients undergo several stages of the chemotherapy to induce and sustain remission; the SMC has approved liposomal cytarabine and daunorubicin for both induction (the first stage) and consolidation (subsequent stages) chemotherapy.

The decision was made following a clinical trial called Study 301, which was run in the USA and Canada. The trial was conducted in adults aged between 60 and 75 years who had “high-risk” AML which had not yet been treated. High-risk AML included patients who had AML as a result of treatment for other cancers (known as therapy-related AML, t-AML) or AML with myelodysplastic changes (AML-MRC). These types of AML can be difficult to treat; for example, therapy-related AML has arisen as a result of other chemotherapy, so one must choose chemotherapy options to which the leukaemia cells will not already be resistant. Patients receiving liposomal cytarabine with daunorubicin had an increased overall survival time of nine months versus just under six months for the comparison group of patients, who were receiving non-liposomal cytarabine with daunorubicin.

SMC have concluded that this medicine is cost-effective for use on the NHS in Scotland, in addition to the evidence presented showing that the treatment works.

“Gaining access to any new treatment that has the potential to increase survival is great news for patients with high-risk AML,” said Zack Pemberton-Whiteley, our Patient Advocacy Director. “The SMC’s announcement is a really positive development for t-AML or AML-MRC patients in Scotland, helping families gain more precious time with their loved ones.”

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