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Lead Researcher Discusses Midostaurin in AML

Shannon Connelly
Published: Friday, May 05, 2017

Richard M. Stone, MD

Richard M. Stone, MD

The recent FDA approval of midostaurin (Rydapt) has excited the field of acute myeloid leukemia (AML). Midostaurin was granted approval for the treatment of adult patients with newly diagnosed FT3-positive AML in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.

, Stone, director of the Adult Leukemia Program, Dana-Farber Cancer Institute, and professor of medicine, Harvard Medical School, discussed the impact that midostaurin could have for patients with AML, as well as ongoing research with the agent.  

OncLive: What impact do you see midostaurin making in the AML community?

Stone: AML is a devastating disease that affects about 15,000 people in the United States annually. Unfortunately, about 40% to 50% of the people who get this disease die of it. It is particularly difficult in older adults who get this disease, and the median age is about 68 to 70, so it is a disease of older adults. But, young people do get it as well. Even in young people, a lot of patients die of it.

-mutant AML.  

What benefit was seen with midostaurin in the phase III RATIFY clinical trial?

This trial had a simple design with the control group getting standard induction chemotherapy, which was daunorubicin and cytarabine, and if those individuals achieved remission, they got standard high-dose post-remission therapy.
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