OBJECTIVES: To compare medical expenditure and effectiveness of different therapies in chronic myeloid leukemia patients who failed in the first-line imatinib treatment. METHODS: A structured questionnaire was designed to collect the detailed data from 28 clinical experts, including treatment alternatives for the patients with chronic myeloid leukemia who failed in first-line imatinib treatment, and medical service utilization, medical expenditure and effectiveness of different therapies (nilotinib, imatinib dose-escalation and allo-SCT). RESULTS: The choices of the second-line therapies for the patients who failed in the first-line imatinib treatment included nilotinib, imatinib dose-escalation and allo-SCT. The cytogenetic response of nilotinib was always higher than imatinib dose escalation. After 1-1.5 years, the cumulative cytogenetic response rate of nilotinib could be as high as 80%, which is significantly higher than that of imatinib. The most common adverse effects of nilotinib included hematologic toxicities (anemia, thrombocytopenia and neutropenia), hyperbilirubinemia, prolongation of QT interval, liver injuries, skin alterations, edema, jaundice and gastrointestinal disturbance. In parallel, bone marrow suppression is the most frequent toxicities of imatinib dose escalation treatment. Other non-hematologic adverse effects were similar, included fluid retention, gastrointestinal disturbance, muscular cramp and bone and joint pain. The expenditure of nilotinib and dose-escalated imatinib therapies was quite similar, and significantly higher than that of first-line imatinib treatment. CONCLUSIONS: It is strongly suggested that nilotinib treatment should be considered as standard treatment of imatinib-failed patients in China.