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Azacitidine not recommended for new AML indication

written by

Leukaemia Care, Charity

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Azacitidine has been assessed by the National Institute for Health and Care Excellence (NICE) and has not been recommended for routine use for the treatment of patients with acute myeloid leukaemia (AML) with more than 30% bone marrow blasts (which refers to the number of abnormal immature white blood cells in a patient’s bone marrow).

The final guidance, published today, details that “Azacitidine is not recommended, within its marketing authorisation, for treating acute myeloid leukaemia with more than 30% bone marrow blasts in people of 65 years or older who are not eligible for haematopoietic stem cell transplant.” It is important to note that this recent decision does not affect those AML patients with 30% bone marrow blasts that are already receiving the drug, who will continue to do so until their clinician feels it appropriate to stop.  

Azacitidine is already recommended for AML patients with 20-30% bone marrow blasts, for intermediate and high risk myelodysplastic syndrome (MDS) patients and for chronic myelomonocytic leukaemia (CMML) with 10–29% marrow blasts. Today’s decision does not prevent patients with these conditions from accessing azacitidine. Access for patients in these settings continues, for both current patients or as a future line of treatment.

Acute myeloid leukaemia is a rapidly progressing form of leukaemia that affects white blood cells known as myeloid cells. There are over 2,660 people diagnosed with AML in England and Wales each year and common symptoms include weakness, tiredness, frequent and reoccurring infections, fever and unusual bruising or bleeding.

The main forms of treatment for AML include chemotherapy and stem cell transplants (SCT), although treatment will vary depending on the specific type of AML, the patient’s age and their general fitness. Sometimes patients are unable to receive typically aggressive chemotherapy or a SCT if they have other conditions (known as comorbidities) that mean it would be difficult for them to withstand this treatment. This can limit the number of treatment options available to such patients and they would usually be treated with best supportive care – which aims to relieve symptoms caused by the leukaemia.

Clinical trials showed that azacitidine showed a prolonged survival benefit when compared with existing, recommended treatments used regularly in the NHS but the data was not statistically significant. This means that it was unclear from the data if the positive results were as a direct result of azacitidine.  Following analysis of this data, NICE were unable to recommend azacitidine for routine use for NHS patients in England and Wales as it could not be considered clinically and cost effective enough.