The addition of the farnesyl transferase inhibitor, tipifarnib, to low dose cytarabine does not improve outcome for older patients with AML


  • Research Funding for this study was provided by Cancer Research UK.

Correspondence: Professor Alan K. Burnett, Department of Haematology, Cardiff University School of Medicine, Heath Park, Cardiff CF14 4XN, UK.



The AML16 trial evaluated the combination of the farnesyltransferase inhibitor, tipifarnib, and low dose cytarabine (LDAC) in older acute myeloid leukaemia (AML) patients in a ‘Pick a Winner’ design. The aim was to double remission rates compared to LDAC, with initial evaluation after 100 patients. Failure to improve remission would result in discontinuation. A total of 65 patients, median age 74 years (range 62–86), were randomized. After reviewing the first 45 patients, the Data Monitoring Committee concluded that the overall aspirations would not be met and recommended closure. The addition of tipifarnib had no effect on response, toxicity or survival.