OPTIMISE-FLT3 optimising therapy in FLT3-mutated acute myeloid leukaemia

Acute myeloid leukaemia (AML) is an aggressive blood cancer and is the commonest form of acute leukaemia in adults, affecting more than 3000 people per year in the UK, the majority of who will die from the disease. Younger and fitter patients can have treatment aiming to cure the disease with cycles of intensive chemotherapy followed for some patients by stem cell transplantation. Survival rates have gradually increased following improvements to chemotherapy, transplantation, better general care measures and the addition of various new targeted drugs for patients in specific AML sub-groups.

This study focuses on a subgroup of AML with mutations in the FLT3 gene, found in about one-third of AML patients. These patients have worse overall outcomes due to increased rates of early disease relapse. Clinical trials in recent years have identified several promising strategies to improve outcomes for patients with FLT3 AML including 1) using an intensified 3-drug chemotherapy protocol called FLAG-Ida 2) adding a drug called midostaurin to inactivate FLT3 and 3) adding a chemotherapy linked-antibody called Gemtuzumab Ozogamicin (also called GO or Mylotarg) but these approaches have not yet been combined in a single trial.