Acute myeloid leukaemia (AML) is one of the most common forms of adult leukaemia.

Most of the approximately 30 percent of AML patients whose leukaemia 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.

This Phase III, multinational, randomised trial analysed 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 randomised 717 adult patients with FLT3-mutated AML to receive either midostaurin (360) in pill form or placebo (357) in addition to standard chemotherapy followed by one 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 eight months compared to only three months in the standard treatment arm.

Standard chemotherapy with midostaurin and one 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 this mutation when added to the standard chemotherapy regimen.

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Source: ASH