Sung Eun LeeSoo Young ChoiSoo Hyun KimSaengsuree JootarHyeoung Joon KimSang Kyun SohnJoon Seong ParkSung Hyun KimDae Young ZangSuk Joong OhDong Wook KimAjou University, School of MedicineKyungpook National University HospitalChonnam National University, College of MedicineFaculty of Medicine, Ramathibodi Hospital, Mahidol UniversityDong-A University, College of MedicineSamsung GroupHallym University, College of MedicineThe Catholic University of Korea2019-08-232019-08-232018-07-01Leukemia Research. Vol.70, (2018), 100-10518735835014521262-s2.0-85048311409https://repository.li.mahidol.ac.th/handle/123456789/45127© 2018 Elsevier Ltd The aim of this study was to investigate the efficacy of nilotinib (NIL) versus high-dose imatinib (IM) versus sustained standard-dose IM for patients with chronic myeloid leukemia (CML) with suboptimal molecular response to first-line IM therapy. Patients with CML who achieved complete cytogenetic response (CCyR) but not major molecular response (MMR) after 18–24 months on first-line IM therapy were enrolled and divided into three treatment cohorts: NIL 800 mg/day (Cohort 1, n = 28) and IM 800 mg/day (Cohort 2, n = 28) in the RE-NICE study, and sustained IM 400 mg/day (Cohort 3, n = 52) in clinical practice. The primary efficacy variable of cumulative rate of MMR by 12 months was not different among the three cohorts. However, the cumulative incidence of MMR by 36 months was significantly higher in Cohort 1 than Cohort 3 (83.1% vs. 57.1%, P = 0.021), but there were no significant differences in Cohort 1 vs. 2 (P = 0.195) and Cohort 2 vs. 3 (P = 0.297). Different profile for adverse events was observed between NIL and high-dose IM therapy. In conclusion, our data suggested that switching to NIL may provide more effective long-term response than sustaining standard-dose IM for patients with suboptimal molecular response to first-line IM.Mahidol UniversityBiochemistry, Genetics and Molecular BiologyMedicineArticleSCOPUS10.1016/j.leukres.2018.06.002