In April 2017, following the results of the RATIFY trial (1), midostaurin, a multikinase FLT3 inhibitor, became the first FDA approved targeted agent for the treatment of acute myeloid leukemia (AML) (2). The addition of midostaurin to standard induction therapy with anthracycline and cytarabine (7 + 3) rapidly became the new standard of care for treatment-naïve, fit patients with FLT3-mutated (FLTmut+) AML (3). More recently, gilteritinib, a selective FLT3 inhibitor, showed superiority to chemotherapy in the treatment of relapsed or refractory FLTmut+ AML (4). With two FLT3 inhibitors now approved by the FDA—that is, the more selective gilteritinib and the less selective midostaurin—the question of which FLT3 inhibitor to use in combination with chemotherapy in the upfront setting has become the subject of much debate (5–7). Leukemia physicians are faced with the choice of using a more selective agent in the front line vs. reserving that agent for the time of relapse. Here, we evaluate the rationale for both approaches.
Keiffer, Gina; Aderhold, Kimberly L; and Palmisiano, Neil D, "Upfront Treatment of FLT3-Mutated AML: A Look Back at the RATIFY Trial and Beyond." (2020). Department of Medical Oncology Faculty Papers. Paper 135.
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