NICE has approved liposomal cytarabine-daunorubicin (Vyxeos) as an option for recently-diagnosed untreated therapy-related acute myeloid leukaemia (AML) and AML with myelodysplasia-related changes in adults.
The decision is based on evidence from the phase 3, multicentre, open-label, randomised Study 301 which included 309 adults aged 60-75 years with high-risk AML.
Treatment with liposomal cytarabine-daunorubicin was associated with increased median overall survival (OS) compared with standard cytarabine and daunorubicin (HR 0.69; 95% CI 0.52-0.90; P=0.005). Median OS was 9.56 months and 5.95 months, respectively.
A post-hoc analysis of the data showed 52 people in the liposomal cytarabine-daunorubicin group and 39 people in the comparator group received a stem cell transplant. In this subgroup, median OS was 10.25 months in the standard cytarabine and daunorubicin group and was not reached in the liposomal cytarabine-daunorubicin group (HR 0.46; 95% CI 0.24-0.89; P=0.0046).
The treatment is administered by intravenous infusion over 90 minutes. For induction of remission, the regimen is daunorubicin 44 mg/m2 and cytarabine 100 mg/m2 on days 1, 3, and 5 for the first course and on days 1 and 3 for subsequent courses, if needed.
For consolidation (5 to 8 weeks after the start of induction), the regimen is daunorubicin 29 mg/m2 and cytarabine 65 mg/m2 on days 1 and 3. A subsequent course of consolidation may be given when there is no disease progression or unacceptable toxicity.