Comparative Effectiveness of Nilotinib and Imatinib for Long-term Outcomes in CML

Nilotinib demonstrated higher cumulative molecular response rates and lower rates of disease progression and death than imatinib in patients with newly diagnosed chronic myeloid leukemia (CML), according to findings published in Leukemia.

To determine the long-term effectiveness of nilotinib vs imatinib, researchers conducted a 10-year follow-up analysis on patients that participated in the phase 3 ENESTnd study (Evaluating Nilotinib Efficacy and Safety in Clinical Trials—Newly Diagnosed Patients).

Patients with newly diagnosed CML in chronic phase were randomized to three groups to receive nilotinib 300 mg twice-daily (n=282), nilotinib 400-mg twice-daily (n=281), or imatinib 400-mg once-daily (n=283).

Follow-up data at 10 years included:

• Cumulative rates of major molecular response and molecular response were higher with nilotinib (300 mg twice daily [BID], 77.7% and 61.0%, respectively; 400 mg BID, 79.7% and 61.2%, respectively) than with imatinib (400 mg once daily [QD], 62.5% and 39.2%, respectively).

• Cumulative rates of [treatment-free remission] eligibility at 10 years were higher with nilotinib (300 mg BID, 48.6%; 400 mg BID, 47.3%) vs imatinib (29.7%).

• Estimated 10-year overall survival rates in nilotinib and imatinib arms were 87.6%, 90.3%, and 88.3%, respectively.

“Nilotinib demonstrated higher cumulative molecular response rates, lower rates of disease progression and CML-related death, and increased eligibility for treatment-free remission,” study authors wrote.

Rates of adverse events were similar among both agents. Treatment with nilotinib yielded higher cumulative rates of cardiovascular events (300 mg BID, 16.5%; 400 mg BID, 23.5%) than treatment with imatinib (3.6%).

“Overall efficacy and safety results support the use of nilotinib 300 mg BID as frontline therapy for optimal long-term outcomes, especially in patients aiming for [treatment-free remission],” researchers concluded. “The benefit-risk profile in context of individual treatment goals should be carefully assessed.”

Reference:
Kantarjian HM, Hughes TP, Larson RA, et al. Long-term outcomes with frontline nilotinib versus imatinib in newly diagnosed chronic myeloid leukemia in chronic phase: ENESTnd 10-year analysis. Leukemia. 2021; 35(2):440-453. doi:10.1038/s41375-020-01111-2