Experimental drug improves survival in high-risk AML patients

Experimental drug improves survival in high-risk AML patients

6 Dec 2015

Midostaurin added to standard chemotherapy is the first targeted treatment to improve survival of a high-risk, genetically defined subgroup of patients with acute myeloid leukemia (AML), reported Richard Stone, MD, of Dana-Farber Cancer Institute, on behalf of the Alliance for Clinical Trials in Oncology group.

The study included AML patients with mutations in the FLT-3 gene

The AML subgroup included patients ages 18 to 60, considered “young adults”, whose cancer cells carried mutations in the FLT-3 gene. The mutated gene drives aggressive growth, so that such patients have a poor prognosis and a high chance of relapse. Treatment with chemotherapy is directed at achieving a remission so that patients can undergo stem cell transplant.

The clinical trial randomized 717 patients to receive standard chemotherapy plus midostaurin, a multi-kinase inhibitor, or chemotherapy plus a placebo, including one year of maintenance therapy. Individuals older than 60 weren’t included in the trial because this chemotherapy regimen is too aggressive for them. With a median follow-up of 57 months, the scientists reported that:

  • Those in the midostaurin arm had a 23% lower risk of dying than those in the placebo group, and
  • They experienced a five-year survival rate of 50.9% versus 43.9% in the placebo arm.


Providing personalized medicine to AML patients

“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,” said Richard M. Stone, MD, director of the Adult Acute Leukemia Program at Dana-Farber.

The study revealed no additional toxicity in the cohort that received midostaurin in addition to chemotherapy. “There was no increase in side-effects in patients assigned to midostaurin compared to those who were assigned placebo,” explained Stone. 

Source: Dana-Farber Cancer Institute

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