Five-Year Results of Nilotinib 400 Mg BID in Early Chronic Phase Chronic Myeloid Leukemia (CML): High Rate of Deep Molecular Response - Update of the Gimema CML WP Trial CML0307

Nilotinib is a potent and selective BCR-ABL inhibitor approved for the frontline treatment of CML based on the results of the phase 3 ENESTnd trial that demonstrated superior efficacy of nilotinib vs. imatinib, with higher and faster molecular responses and lower rates of progressions to accelerated-blastic phase (AP/BP)(3 years of follow-up). In the IRIS trial, after 5 years of follow-up, 69% of patients were still on imatinib; the event-free survival (EFS) and progression-free survival (PFS) were 83% and 93%, respectively (Druker BJ et al, NEJM 2006). In an intention-to-treat analysis of patients treated frontline with imatinib at the Hammersmith Hospital, EFS at 5 years was 63% and PFS was 83% (de Lavallade H, J Clin Oncol 2008). The long-term evaluation of the patients treated with nilotinib frontline is extremely relevant. METHODS: The GIMEMA CML WP conducted a multicentre phase 2 trial with nilotinib 400mg BID as frontline therapy (ClinicalTrials.gov.NCT00481052). Median follow-up for the present analysis was 51 months (range: 48 – 58 months); five years median follow-up data will be presented. Definitions: MR3.0 (Major Molecular Response) as a BCR-ABL/ABL ratio <0.1% IS; MR4.0, BCR-ABL/ABL ratio