In patients with FLT3-mutated acute myeloid leukemia (AML), maintenance therapy with midostaurin after intensive chemotherapy plus midostaurin appeared to be safe and was associated with a benefit for some patients over watch-and-wait, according to a recent study. The study results were presented in a poster at the Annual Meeting of the Society of Hematologic Oncology (SOHO) by Adolfo de la Fuente, MD, of MD Anderson Cancer Center Madrid in Madrid, Spain, and colleagues.

“The standard of care for FLT3-AML patients has been for decades intensive chemotherapy and consolidation with allo-stem cell transplantation,” Dr de la Fuente said in an audio presentation accompanying the research team’s poster. Midostaurin has become an approved treatment in combination with intensive chemotherapy for patients with FLT3-mutated AML, but effects with midostaurin maintenance have not been extensively explored.

In this real-world, retrospective, multicenter study based in Spain, Dr de la Fuente and colleagues examined outcomes with midostaurin maintenance in comparison with both allogeneic stem cell transplantation (allo-SCT) and a watch-and-wait approach. Included patients were adults with FLT3-mutated AML who had achieved complete remission after intensive chemotherapy plus midostaurin. The researchers evaluated outcomes related to response, toxicity, and overall survival (OS) and according to European LeukemiaNet risk status.

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There were 175 patients included in the study. Patients had a median age of 53 years, and 111 of the patients were 60 years of age and older. There were 110 patients with an Eastern Cooperative Oncology Group performance status of below 2.

The rate of complete remission in this population was 81.4%. The median OS in this study has not yet been reached, and the 2-year OS rate for this population was 68%. Among patients with intermediate-risk status, there was no significant difference in OS with receipt of maintenance midostaurin compared with allo-SCT (P =.7). However, patients with low- and intermediate-risk status appeared to experience a significant OS benefit with midostaurin maintenance in comparison with a watch-and-wait approach (P =.01).

In terms of safety, there was 1 reported case of QT prolongation that required discontinuation of midostaurin. Deaths and febrile neutropenia reportedly did not occur in relation to midostaurin maintenance.

Dr de la Fuente and colleagues concluded that with these data the safety profile of midostaurin maintenance was confirmed in patients with FLT3-mutated AML who had reached complete remission following intensive chemotherapy plus midostaurin. The researcher team considered maintenance midostaurin to show a significant benefit in comparison with watch-and-wait for patients with low- to intermediate-risk status. They also concluded that they did not see OS differences with midostaurin maintenance compared with allo-SCT for patients with intermediate risk.


de la Fuente A, Diaz Beya M, Beneit P, et al. Midostaurin maintenance versus allo SCT versus W&W in FLT3-mutated AML: a “real-life” multicenter study. Presented at SOHO 2022; September 28-October 1, 2022; Abstract AML-122.