NICE have published a positive Final Appraisal Document recommending routine NHS use of venclyxto (venetoclax) with azacytidine for the treatment of aggressive blood cancer acute myeloid leukaemia (AML). Approximately 3,200 people are diagnosed with AML in the UK every year, equalling more than eight new cases every day. The treatment is recommended as an option for untreated AML in adults when intensive chemotherapy (IC) is unsuitable.

NICE’s decision to authorise the combination offers patients a treatment option with significantly improved overall survival, increased rates of rapid and durable remission and blood transfusion independence, compared to treatment using azacitidine alone.

AML is an aggressive and difficult-to-treat blood cancer with a low survival rate. The causes of AML are often unclear, although factors raising risk include previous exposure to very high levels of radiation, including previous radiotherapy treatment, previous chemotherapy, having a blood disorder or some genetic conditions, such as Down’s syndrome, and smoking. The disease predominately affects older people, and around 40% of those newly diagnosed are unable to tolerate the standard first-line treatment of IC on account of health issues and presence of other conditions.

Median survival for AML is just 6-10 months. During this period patients often require hospitalisation and frequent blood transfusions, resulting in a significant impact on quality of life.

Venetoclax treatment can be taken at home, reducing patient dependency on blood transfusions and potentially meaning fewer hospital visits for the clinically vulnerable patients who are more susceptible to coronavirus infections, as well as others. The treatment is being developed by AbbVie and Roche.

Professor Charlie Craddock, academic director of the Centre for Clinical Haematology, Queen Elizabeth Hospital, Birmingham and professor of haemato- oncology, at the University of Birmingham, shared: “Venetoclax plus azacitidine is the most important advance in the treatment of AML for patients not suitable for intensive chemotherapy in over 30 years and represents a step change in therapy for this common blood cancer”.