Sorafenib for the treatment of younger AML patients

This study examined the effectiveness of sorafenib (Nexavar) in acute myeloid leukemia (AML) patients younger than age 60. The study concluded that sorafenib was effective but increased negative side effects.

Targeted therapies are treatments that block certain proteins involved in cancer growth. Sorafenib is a targeted therapy that blocks multiple proteins involved in AML growth. Sorafenib combined with intensive chemotherapy was not effective in older AML patients (over age 60), and increased the rate of serious side effects. Younger patients are more able to withstand intensive treatments. Proteins involved in cancer growth can also differ between older and younger AML patients. Sorafenib may be an effective treatment option for younger AML patients.

This study explored the effectiveness and safety of sorafenib in AML patients 60 years old or younger. The study included 267 patients. 134 patients were randomly assigned to sorafenib treatment. 133 patients were assigned to placebo treatment (substance with no active effect). All patients were also treated with chemotherapy. Low-risk patients were also treated with chemotherapy and sorafenib or placebo for consolidation and maintenance (treatments to maintain remission). Higher risk patients could undergo stem cell transplantation. Patients were followed for an average of 3 years.

Average event-free survival (time until treatment failure, relapse, or death) was 21 months in the sorafenib group. This was compared to 9 months in the placebo group. Average 3-year event-free survival rates were 40% in the sorafenib group and 22% in the placebo group. There was no significant difference in overall survival (time from treatment until death from any cause).

Serious or severe side effects were more common in the sorafenib group. Patients treated with sorafenib had a 54% higher risk of fever, a 7.89-fold higher risk of diarrhea, and a 3.75-fold higher risk of bleeding. Risk of skin reactions and cardiac (heart) events was also increased.

This study concluded that sorafenib was an effective treatment for younger AML patients, but was associated with increased negative side effects.

This study was funded by Bayer HealthCare, the manufacturer of sorafenib.