DefinePK

Abstract

Objective: To compare the efficacy, safety, and progression-free survival (PFS) of nilotinib versus ponatinib as second-line therapy in chronic-phase chronic myeloid leukemia (CP-CML) patients resistant or intolerant to imatinib and negative for the T315I mutation.
Methodology: A single-center retrospective -prospective cohort study was conducted between January 2008 and 2023, enrolling 35 CP-CML patients resistant or intolerant to first-line imatinib and confirmed negative for T315I mutation. Patients received nilotinib or ponatinib per clinical guidelines. The primary outcomes were major molecular response (MMR) rates and PFS, analyzed using Kaplan–Meier survival curves with log-rank testing. Adverse events were recorded according to standardized criteria.
Results: Nineteen patients received nilotinib and sixteen received ponatinib. Baseline risk scores were comparable (p=0.491). After second-line treatment, MMR was achieved in 15.8% of nilotinib patients and 37.5% of ponatinib patients (p=0.245). PFS showed a numerical trend favoring ponatinib, but without statistical significance (p=0.743). No deaths occurred during the observation period. Adverse events included mild allergic reactions in both groups, with thyroiditis observed only in nilotinib-treated patients and dermatitis in ponatinib-treated patients.
Conclusions: Both nilotinib and ponatinib demonstrated durable disease control as second-line therapies in imatinib-resistant or intolerant CP-CML patients negative for T315I, with acceptable safety profiles. While ponatinib showed a trend toward improved PFS, differences were not statistically significant.