Nilotinib may be a promising frontline therapy for patients with chronic myeloid leukemia in chronic phase (CML-CP), according to study results reported in the British Journal of Haematology.

In this observational, multicenter, prospective study conducted in France, 183 patients with newly diagnosed CML-CP were evaluated for outcomes after 24 months of treatment with nilotinib.

The primary end point was undetectable molecular disease at a threshold of 4-log sensitivity (uMR4) at 24 months, assessed by determining a patient’s transcript levels of BCR-ABL1 relative to a standardized level. Transcript levels were analyzed by quantitative polymerase chain reaction.

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Other assessments included safety, treatment adherence measured with the Morisky Green Levine Medication Adherence Scale, and quality of life (QoL) using the EORTC QLQ-C30 and EQ-5D-3L questionnaires.

Nilotinib was discontinued before 24 months by 53 patients (29%); discontinuation was attributed to intolerance in 29 patients (15.8%) and lack of efficacy in 19 patients (10.4%).

Molecular evaluations were completed for 112 (91.8%) of the 122 patients still taking nilotinib at 24 months. A major molecular response was seen in 95.5% of evaluated patients, and 32.1% of evaluated patients reached uMR4.

QoL overall was fairly high and stable throughout the 24 months of follow-up, showing some improvements with nilotinib therapy. At 24 months, 94 patients were evaluated for treatment adherence, 77.7% of whom showed full treatment adherence.

Safety was assessed in all 183 patients, and 70.5% reported adverse events. Pruritis (14.8%) and asthenia (13.7%) were the most common adverse events associated with nilotinib. Seven patients had 1 or more cardiovascular ischemic events.

“Nilotinib is associated with a high adherence level and maintenance of a good QoL,” the authors wrote in their report. “Hence, this study supports the use of nilotinib as a first-line treatment option for patients with CML-CP.”


  1. Huguet F, Cayuela JM, Cambier N, et al. Nilotinib efficacy, safety, adherence and impact on quality of life in newly diagnosed patients with chronic myeloid leukaemia in chronic phase: a prospective observational study in daily clinical practice [published online August 8, 2019]. Br J Haematol. doi:10.1111/bjh.16145