Chronic Myeloid Leukemia Coverage from Every Angle

Long-Term Efficacy of Nilotinib in Chronic Phase CML

By: Lauren Harrison, MS
Posted: Wednesday, March 18, 2020

The second-generation tyrosine kinase inhibitor nilotinib showed long-term efficacy in patients with chronic phase chronic myeloid leukemia (CML), as many patients (75%) achieved a sustained molecular response level of 4.5. Hagop Kantarjian, MD, of The University of Texas MD Anderson Cancer Center, and colleagues published this work in Cancer.

“The most noticeable immediate benefit is the achievement of an early molecular response…. This provides the possibility for patients to attempt treatment-free remission in the future,” concluded the authors.

The single-institution phase II study recruited 122 patients with newly diagnosed chronic phase CML. Patients received 400 mg of nilotinib twice daily, and the median follow-up was 78.3 months. At the last follow-up, 56% of the patients remained on the therapy.

The complete cytogenetic response rate and the molecular response rate were 91% each, and 75% of patients achieved a greater than 4.5-log reduction in BCR-ABL transcripts (MR4.5). Approximately 59% of patients sustained MR4.5 at 2 years. The event-free survival at 5 years was 89%, and at 10 years, it was 85%; the overall survival rate was 93% at 5 years and 88% at 10 years. Four patients (3%) experienced disease progression to accelerated or blast phase and seven patients (6%) died while on the study.

Treatment was discontinued in 19% of patients enrolled in the trial due to toxicity. The most common adverse events leading to treatment discontinuation were cardiovascular events (10%) and biochemical abnormalities (6%). Ischemic cardiovascular events occurred in 8% of patients. Common nonhematologic adverse effects were rash (55%), elevated bilirubin levels (57%), and elevated aminotransferase levels (48%).

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