Liposomal cytarabine and daunorubicin approved by NICE

The National Institute of Health and Care Excellence (NICE) have today recommended the use of liposomal cytarabine with daunorubicin to treat previously untreated therapy-related AML and AML with myelodysplastic changes.

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, and so cells replace the cytidine with the 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 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; NICE have 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 and 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) or AML with myelodysplastic changes. These types of AML are 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 9 months versus just under 6 months for the comparison group of patients, who were receiving non-liposomal cytarabine with daunorubicin.

NICE has also concluded that the treatment is within the reasonable cost per quality-adjusted life year (QALY); this is a measure of cost-effectiveness, where treatments are decided for approval based on how much they cost in order to give patients one extra year of life.

“A diagnosis of acute myeloid leukaemia can have a huge emotional impact on the lives of patients, as well as their family and friends,” said Zack Pemberton-Whiteley, Patient Advocacy Director at Leukaemia Care. “We welcome the decision to recommend this treatment for adults with high-risk AML. Leukaemia Care has been working with NICE to enable patients to access this important new treatment, where options have been previously limited.”

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