Journal of Blood Disorders & Transfusion

ENESTgoal: Evaluation of Treatment-Free Remission (TFR) in patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) and sustained deep Molecular Response (MR) following switch to nilotinib

2^nd International Conference on Hematology & Blood Disorders

September 29-October 01, 2014 DoubleTree by Hilton Baltimore-BWI Airport, USA

Andrea Magley, Eric J Feldman, Michae W Deininger, Jerald Radich, Christopher Keir, Felice Lin and Michael Mauro

Posters: J Blood Disorders Transf

Abstract:

Sustained TFR has been reported in clinical trials ofpatients withCML-CP who achievedsustaineddeep MR on imatinib therapy. Nilotinib treatmentresults in high rates of sustained deep MR, a key eligibility criterion for TFR studies.The randomized, phase 2 ENESTgoal study(NCT01744665) will evaluate TFR among patients with CML-CP who achievesustained MR4.5 (BCR-ABL≤0.0032% on the International Scale [IS] by real-timequantitative polymerase chain reaction [RQ- PCR]) after switching tonilotinib.ENESTgoal will enrollapproximately 300 patients with major molecular response (BCR- ABLIS≤0.1%)but with detectable disease (BCR-ABLIS>0.0032%), as assessed by a central laboratory, after ≥1 year ofimatinib therapy. Patients will switch to nilotinib 300 mg twice dailyonstudy entry andbe monitored quarterly for up to 2 years; those who achieveconfirmed MR4.5 during the monitoring phase will be randomized to enter a 1- or 2-year consolidation phaseof continuednilotinib treatment. Patients will then stop nilotinib therapy and enter the 3-year TFR phase if MR4.5is maintained throughout the consolidation phase. RQ-PCR assessments will be performed monthly for the first 6 months of the TFR phase, then every 2 months for 18 months and then quarterlythereafter. Patients with molecular relapse, defined in ENESTgoalas confirmed loss of MR4 (or BCR-ABLIS>0.01%), will re-initiate nilotinibtherapy. The primary endpoint of the study is the molecular relapse-free rate at 6 months after suspension of nilotinib therapy. ENESTgoal is currently open for enrollment in the United States.