Leukemia: Acute Myeloid Leukemia (AML)

    Actionable Target Abnormality Clinical Experience with Targeted Agent
    FLT3 Internal tandem duplication (ITD) Sorafenib (VEGFR inhibitor):
    • Compassionate use, monotherapy: Median time from allo-SCT to relapse = 192 d (1)
    • Phase 2, with idarubicin + cytarabine: CR 92%, CR with incomplete platelet recovery (CRp) 8% (2)
    • Phase 2, with azacytidine: CR 16%, PR 3%, CR with incomplete count recovery (CRi) 27%; median duration of CR/CRi 2.3 mo (3)
    Midostaurin (PKC-α/VEGFR2/c-KIT/PDGFR/FLT3 inhibitor):
    • Phase 3, 7 + 3 ±midostaurin in pts with FLT3 mutated AML: CR rates similar o] vs. 54%), but significant improvement in OS (
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