Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts – guidance (TA399)

Azacitidine is NOT recommended 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 because of concerns with its clinical as well as cost-effectiveness.

During the appraisal, the committee found:

• The clinical trial demonstrated gains favouring azacitidine in its primary efficacy end point of overall survival, but failed to reach statistical significance when comparing azacitidine with the combined conventional care regimen.

• The committee concluded that the degree to which azacitidine was more effective than any of the individual conventional care regimens was very uncertain.

• The most plausible incremental cost-effectiveness ratio for azacitidine compared with a conventional care regimen is £240,000 per quality-adjusted life year gained.