Long-term nilotinib treatment for newly diagnosed chronic-phase chronic myeloid leukemia

This study aimed to investigate if nilotinib was safe and effective as a long-term treatment in patients with chronic-phase chronic myeloid leukemia.  

This study concluded that nilotinib is safe and effective in the long-term in these patients.  

Nilotinib (Tasigna) is a type of targeted therapy known as a tyrosine kinase inhibitor (TKI). Nilotinib has been approved as frontline and salvage therapy for patients with chronic-phase (CP) chronic myeloid leukemia (CML). However, the long-term safety and effectiveness of nilotinib as a long-term treatment for patients with CP-CML remain under investigation.  

This study involved 122 patients with newly diagnosed CP-CML. Patients received 400mg nilotinib twice a day. Patients were followed for an average of 78.3 months. 

The complete cytogenic response (CCyR) rate was 93%. CCyR is when no cells with Philadelphia (Ph+) chromosome are found. Ph+ are found in cancer cells. The major molecular response (MMR) rate was 91%. MMR is a low level of BCR-ABL gene found in cells. High levels of BCR-ABL are found in cancer cells.  

The estimated event-free survival (EFS; survival without complications related to CML) rate at 5 years was 89%. The estimated EFS rate at 10 years was 85%. The estimated overall survival (OS) rate at 5 years was 93%. The estimated OS rate at 10 years was 88%.  

19% of patients ended treatment mainly due to cardiovascular (heart-related) side effects and biochemical abnormalities. 55% of patients experienced a rash as a side effect. 57% experienced elevated bilirubin (can cause jaundice) and 48% experienced elevated liver enzymes (may indicate liver disease).  

3% of patients progressed to accelerated or blast phase while being treated and 6% of patients died during the study. 56% of patients remained on treatment after the last follow up. 

This study concluded that nilotinib is safe and effective for long-term treatment of patients with CP-CML.  

This study had a relatively small number of participants. Larger studies are needed.