Novel Targeted Therapy Improves Survival in Acute Myeloid Leukemia

An experimental targeted therapy was found to offer a survival advantage over placebo in the treatment of acute myeloid leukemia (AML) during a recent clinical trial.

The addition of the multi-kinase inhibitor midostaurin to standard chemotherapy is the first targeted treatment to improve survival in a high-risk subgroup of patients with AML, according to the Dana-Farber Cancer Institute.

"We were pleased to learn that patients who had midostaurin added to their therapy survived more commonly and longer than those who received placebo," said Richard M. Stone, MD, director of the Adult Acute Leukemia Program at Dana-Farber.

In data presented the 57th American Society of Hematology Annual Meeting and Exposition in Orlando, FL, researchers evaluated the drug in a subgroup of patients aged between 18 to 60 with cancer cells that carry mutations in the FLT-3 gene.

This genetic mutation has been found to cause aggressive growth, which leads to a poor prognosis and greater chance of relapse. Patients with AML are administered chemotherapy in hopes of causing remission to allow for a stem cell transplant.

The CALGB 10603/RATIFY trial randomized 717 patients to receive either standard chemotherapy plus midostaurin or chemotherapy with placebo, followed by 1 year of maintenance therapy.

After a median follow-up of 57 months, patients administered midostaurin were found to have a 23% lower death risk than patients receiving placebo. Meanwhile, patients in the midostaurin group had a 5-year survival rate of 50.9% compared with 43.9% in the placebo group.

"This trial is the first step in applying the theories of personalized medicine to patients with AML, specifically those patients with AML who have a FLT-3 mutation who we have shown are likely to benefit from the addition of this targeted agent, midostaurin, to standard chemotherapy," Dr. Stone said.

Furthermore, there were no additional toxicity risks observed in the midostaurin group.

"There was no increase in side-effects in patients assigned to midostaurin compared to those who were assigned placebo," Dr. Stone said. "This may have been because the side-effects of chemotherapy dwarfed any that might have been attributed to the midostaurin."