Long-term outcomes for patients with acute myeloid leukemia treated with sorafenib

This study aimed to investigate the long-term outcomes of sorafenib (Nexavar) treatment and maintenance therapy in patients with acute myeloid leukemia (AML).  

This study concluded that the addition of sorafenib to chemotherapy led to better outcomes for these patients.  

Sorafenib is a type of targeted therapy called a tyrosine kinase inhibitor (TKI). It can be used in the treatment of some types of cancer, including acute myeloid leukemia (AML). Early results of a study of sorafenib in newly diagnosed patients with AML showed that sorafenib led to a significant improvement in survival without relapse or complication from AML. However, the longer-term outcomes of sorafenib are still unknown. 

This study involved 267 patients with newly diagnosed AML. 134 patients received sorafenib and 133 received a placebo as induction therapy or maintenance treatment. All patients also received standard chemotherapy. Induction is the first line of treatment. Maintenance treatment aims to prevent relapse or progression of the disease. Patients were followed up for an average of 78 months.  

After 5 years, 41% of the sorafenib group were event-free (no complications from AML). This was compared to 27% for the placebo group. 53% of the sorafenib group and 36% for the placebo group were relapse-free after 5 years. Sorafenib was associated with a 36% lower risk of relapse compared to placebo. The overall survival rate after 5 years was 61% in the sorafenib group and 53% in the placebo group. 

In patients who relapsed, 88% received an allogeneic stem cell transplantation (alloSCT). AlloSCT uses stem cells from a donor to replace damaged cells. Four years after alloSCT, the rate of relapse was 54% for the sorafenib group compared to 35% for the placebo group. 

This study concluded that the addition of sorafenib to chemotherapy as induction and maintenance treatment led to better survival without complications and relapse in patients with AML.  

This study did not include data about side effects. Further studies are needed.