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.
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.”
- 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