Venetoclax in combination with a hypomethylating agent or low dose cytarabine for newly diagnosed acute myeloid leukaemia

Venetoclax is being developed for newly diagnosed patients with acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy. AML is an aggressive type of blood cancer that starts from certain types of young white blood cells called granulocytes or monocytes in the bone marrow. AML usually develops over a few weeks and becomes increasingly more severe. If left untreated it would cause death within a few weeks or months. High intensity chemotherapy followed by haematopoietic stem cell transplant (HSCT) is the standard treatment for AML in fit patients and is often very demanding with potentially severe side effects. Patients who cannot tolerate or who do not wish to receive high intensity chemotherapy are given low dose cytarabine or a hypomethylating agent (azacitidine or decitabine). Decitabine is not recommended for use by NICE however.
Venetoclax blocks the action of a specific protein which is present in high amounts in AML cells. This results in the death of the cancer cells and thereby slows the progression of the disease. Venetoclax is already licensed to treat other types of leukaemia and is thought that it might be of significant benefit for patients with AML when used in combination with other medicines in patients who cannot receive standard high dose treatment. Venetoclax in combination with a hypomethylating agent (azacitidine or decitabine) or low dose cytarabine may offer a first-line treatment option for newly diagnosed patients with AML.