Long-term outcomes of nilotinib treatment for chronic myelogenous leukemia patients with imatinib resistance or intolerance / 南方医科大学学报

ABSTRACT

<p><b>OBJECTIVE</b>To evaluate the long-term clinical efficacy and safety of nilotinib in the treatment of chronic myelogenous leukemia (CML) patients with imatinib resistance or intolerance.</p><p><b>METHODS</b>Twenty-six CML patients with imatinib resistance or intolerance received nilotinib treatment at the dose of 400 mg once or twice daily. The patients were followed up for nearly 5 years with regular monitoring of the hematologic, cytogenetic and molecular biological markers and recording of the clinical manifestations and biochemical indicators to evaluate the therapeutic effect and adverse events.</p><p><b>RESULTS</b>The median duration of nilotinib therapy was 17 (1-56) months, and the patients were follow up for a median of 51 months. At the last follow-up, 16 (61.5%) patients achieved a complete hematologic response, 13 (50.0%) achieved a major cytogenetic response, 9 (34.6%) achieved a complete cytogenetic response, and 7 (26.9%) achieved a major molecular response accumulatively. Nonhematologic adverse events were mostly of grade l or 2. The most common adverse effects possibly related to nilotinib were increased bilirubin (69.2%) and rash (57.7%). Grade 3 or 4 hematologic adverse events included thrombocytopenia (53.8%), neutropenia (26.9%) and anemia (19.2%). The patients in chronic and remission phase had better efficacy and fewer hematological side effects than those in advanced phase.</p><p><b>CONCLUSION</b>Nilotinib is an effective and safe treatment option for imatinib-resistant or -intolerant CML patients, especially for those in chronic and remission phase.</p>