Leukaemia Care successfully campaigns for new acute myeloid leukaemia (AML) treatment in Scotland

Exciting news for acute myeloid leukaemia (AML) patients in Scotland. It has been announced that venetoclax with a hypomethylating agent (e.g., azacitidine) will be a treatment option for newly diagnosed AML patients in Scotland who are unsuitable for intensive chemotherapy.

What’s the news?

Today, we welcome the news that a new treatment, venetoclax with a hypomethylating agent (e.g., azacitidine), has been recommended by Scottish Medicines Consortium (SMC) as a treatment option for newly diagnosed acute myeloid leukaemia (AML) patients in Scotland who are unsuitable for intensive chemotherapy. This comes following NICE’s approval of this treatment in the same group of patients in England earlier this year. You can find out more about this announcement here.

How was Leukaemia Care involved in this approval? 

We at Leukaemia Care aim to be involved in every appraisal of treatments for leukaemia, myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPNs) in all nations of the UK. Medicines are essential to improving survival of leukaemia patients. New medicines are even more important to AML patients who cannot have intensive chemotherapy because the existing treatment options are not very effective. 

For this appraisal, we consulted patients who had experience of the treatment to better understand why this would be a useful treatment option for them, as well as what the issues are with existing treatments. We submitted this information to the SMC in a patient group submission, making a case for the treatment to be approved. We also attended follow up meetings and described the experience of patients who cannot have intensive chemotherapy, ensuring  that the patients who would be affected by the approval of this treatment were being represented and heard.

Which group of patients can access this new treatment?

This is a treatment for newly diagnosed AML patients who are unsuitable for intensive chemotherapy. 

People can be unsuitable for chemotherapy for a number of reasons. Chemotherapy is an intensive treatment with sometimes severe side effects, so clinicians must discern if the treatment is likely to make patients better rather than worse. Patients for whom the side effects might outweigh the benefits of chemotherapy can include those with comorbidities and elderly patients. However, intensive chemotherapy is so intensive because it needs to put patients into remission effectively. Therefore, those who cannot have intensive chemotherapy must be treated with medicines that are much less effective, reducing their chance of experiencing a long term remission.

Why has the SMC made this recommendation?

Before this, there were limited treatment options for those who are newly diagnosed with AML and who are unsuitable for intensive chemotherapy treatments. Treatment in this cohort was restricted to azacitidine, low dose cytarabine and daunorubicin (LDAC) or having treatments which manage symptoms rather than treat the disease. These options are not very effective at improving survival of patients, and most patients experience relapse quickly. This treatment has already been approved for use on the NHS in England for the same group of patients, so we are pleased that the SMC recommends it for the same use in Scotland. 

This treatment is particularly welcomed given that in the last few years there have been a breakthrough in treatment options for AML for those who were fit enough for intensive treatments For example, there have been a number of treatments targeting specific genetic mutations (e.g. FLT3-ITD inhibitors) and improved formulations of intensive chemotherapy. Yet very few treatment options have come about for those not fit enough intensive chemotherapy, which is why we welcome today’s decision by the SMC.  

What are the benefits of this treatment for patients?

Venetoclax has performed well at clinical trials; it has an improved overall survival rate compared to the current standard treatment options for those who cannot tolerate intensive treatments. The clinicians we spoke to consider this a significant improvement in how they treat these patients.

A patient we spoke to who had taken venetoclax with azacitidine said that they were able to continue carrying out all of their usual duties, including driving, cooking, housework, exercise etc. This suggests that the side-effects of this treatment are still manageable for this patient, which is the aim of non-intensive chemotherapy. 

Venetoclax with azacitidine is administered as an oral tablet which is the most popular route of delivery among AML patients respondents in the Leukaemia Care survey. This is likely because oral medicines can be taken by the patient at home and reduce travel to and time spent in hospital, which undoubtedly also has a positive impact on a patient’s finances. 

When will the treatment be available on the NHS to eligible patients in Scotland? 

The treatment will be available as soon as your local NHS board has included it in the board’s local formulary. Here is where you can find out if your local board has accepted the treatment: 

How does this combination of drugs work? 

Acute myeloid leukaemia (AML) is a type of leukaemia that develops rapidly and is life threatening if not treated quickly. White blood cells are important for fighting infection in your body, but they must develop properly in your blood to do this. Leukaemia occurs when you produce too many immature cells that cannot work properly to fight off infection. The immature AML cells also stop you making other blood cells you need, like red blood cells.

Venetoclax is an oral BCL-2 inhibitor. The BCL-2 protein prevents programmed cell death, the normal way the body gets rid of cells that have not developed properly. If BCL-2 protein is present in your cells, the leukaemia cells can survive. Venetoclax blocks this activity of the BCL-2 protein, causing cell death of the leukaemia cells. 

Ventoclax is used with hypomethylating agents. These drugs work by preventing a chemical reaction, called methylation, happening to DNA. When DNA cannot be methylated, it is a sign to the cell that the DNA must be destroyed. This in turn triggers the leukaemia cells to die too.

Two types of hypomethylating agents used for AML are azacitidine and cytarabine; which one patients would have would depend on the amount of leukaemia cells in their body at the time of treatment. However, neither of them are effective for very long and still bring some side effects. The combination of venetoclax with azacitidine has been shown to work better. Venetoclax is also being looked at in combination with cytarabine, but this assessment is still ongoing.


If you have any questions about this blog, please contact our advocacy team on advocacy@leukaemiacare.org.uk

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