APL0406

The Italian-German APL0406 trial randomised adults with newly diagnosed acute promyelocytic leukaemia and a white cell count of 10 x 10^9/L or less to all-trans retinoic acid plus arsenic trioxide for induction and consolidation, or to all-trans retinoic acid plus idarubicin induction followed by anthracycline-based consolidation and maintenance. The primary endpoint was event-free survival at two years, tested for non-inferiority.

The chemotherapy-free arm was not only non-inferior but superior for event-free and overall survival, with fewer infections and deaths in remission, and the advantage held in the extended analysis with more patients. Retinoic acid plus arsenic became the worldwide standard for non-high-risk disease, the first cure of a leukaemia without cytotoxic chemotherapy.