ASH 2015: Midostaurin Improves Survival in Patients With FLT3-Mutated Acute Myeloid Leukemia Aged 18–60
A study presented by Stone et al at the 57th American Society of Hematology (ASH) Annual Meeting described a phase III trial of the first targeted therapy for genetically defined subset of patients with acute myeloid leukemia and its improvement of their survival (Abstract 6).
Acute myeloid leukemia (AML) is one of the most common forms of adult leukemia. Most of the approximately 30% of AML patients whose leukemia cells have a mutation in the FLT3 gene have a particularly poor prognosis, as their disease tends to be more aggressive and is associated with a higher incidence of relapse. While targeted therapies have improved treatment for other blood cancers, there have been few advances in AML.
Midostaurin is an experimental drug that inhibits many enzymes, including mutant FLT3.
Study Findings
This phase III, multinational, randomized trial analyzed whether adding midostaurin to standard chemotherapy would improve survival when compared to standard chemotherapy alone in adults aged 18 to 60 with this mutation.
Investigators randomly asssigned 717 adult patients with FLT3-mutated AML to receive either midostaurin (n = 360) in pill form or placebo (n = 357) in addition to standard chemotherapy, followed by 1 year of maintenance therapy with the new drug.
The median time to either failure to achieve remission, relapse, or death in patients who received midostaurin was 8 months, compared to only 3 months in the standard treatment arm.
Standard chemotherapy with midostaurin and 1 year of maintenance therapy significantly improved median overall survival (74.7 months compared to 26.0 in the group receiving only standard therapy).
These findings suggest that midostaurin improves outcomes in younger adults with AML with the FLT3 mutation when added to the standard chemotherapy regimen.
The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
