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Long-term results of a phase 2 trial of nilotinib 400 mg twice daily in newly diagnosed patients with chronic-phase chronic myeloid leukemia

Cancer Feb 07, 2020

Masarova L, Cortes JE, Patel KP, et al. - Researchers assessed long term outcomes [with a median follow-up of 78.3 months (interquartile range, 58.4-96.5 months] among patients (n = 122) with newly diagnosed chronic-phase chronic myeloid leukemia (CP-CML) treated with nilotinib (second-generation inhibitor of BCR-ABL1 tyrosine kinase) 400 mg twice daily, in this single-institution, phase 2 study. The complete cytogenetic response rate, as well as the major molecular response (MR) rate, was estimated to be 91%. The achievement of a ≥ 4.5-log decrease in BCR-ABL1 transcripts (MR4.5) and a sustained MR4.5 beyond 2 years was reported in 75% and 59% of patients, respectively. At 5 years, event-free survival and overall survival rates were reported to be 89% and 93%, respectively, and the respective corresponding rates noted at 10 years were 85% and 88%. Rash (55%), high bilirubin (57%), and raised aminotransferases (48%) were documented as the top 3 nonhematologic toxicities. Transient and mild hematologic toxicity was seen. Overall, nilotinib 400 mg twice daily was proved to have long-term efficacy in patients with CP-CML. The achievement of sustained MR4.5 by a majority of patients is possible.
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