Long term outcomes of nilotinib versus imatinib in chronic phase chronic myeloid leukemia

This study aimed to investigate the long-term outcomes of nilotinib (Tasigna) versus imatinib (Gleevec) in patients with chronic-phase chronic myeloid leukemia (CP-CML). 

This study concluded that nilotinib provided good long-term outcomes for these patients.  

CML results from a gene abnormality where the abnormal BCR-ABL gene results in CML growth and spread. Nilotinib and imatinib are a type of targeted therapy called a tyrosine kinase inhibitor (TKI) that targets the BCR-ABL gene. It is used for the treatment of CML. 

Previously, it was shown that nilotinib resulted in higher rates of major molecular response (MMR) at 5 years compared to imatinib. MMR is when there is little or no BCR-ABL gene found in a patient's blood. However, it was unknown if these benefits last for longer in patients with CML.  

This study involved 305 patients newly diagnosed with CP-CML. They received treatment with nilotinib 300mg twice daily (group 1) or 400mg twice daily (group 1) or with imatinib 400mg once daily (group 3). Patients were followed for 10 years or more to compare the long-term outcomes of nilotinib and imatinib treatment.  

The 10-year rate of MMR was 77.7% for group 1, 79.7% for group 2, and 62.5% for group 3. Deep molecular response (MR4.5) is when the level of the BCR-ABL gene is minimal and is a sign of remission. The 10-year MR4.5 rate was 61% for group 1, 61.2% for group 2, and 39.2% for group 3.  

After 10 years, 48.6% in group 1 and 47.3% in group 2 were eligible for treatment-free remission (TFS). This was compared to 29.7% in group 3. The estimated 10-year overall survival rates were 87.6% for group 1, 90.3% for group 2, and 88.3% for group 3.   

The overall frequency of side effects was similar with nilotinib and imatinib. At 10-years, the rate of heart-related side effects was 16.5% in group 1, 23.5% in group 2, and 3.6% in group 3.  

This study concluded that nilotinib is safe and effective for optimal long-term outcomes in patients with newly diagnosed CP-CML.  

This study was supported by Novartis, the manufacturer of nilotinib.