Advancing Targeted Therapy in Acute Myeloid Leukemia (AML) with Quizartinib

About

Acute myeloid leukemia (AML) remains a clinically challenging hematologic malignancy. Advances in molecular profiling have transformed AML management, shifting from a standard chemotherapy-based approach toward precision medicine. Mutations in Feline McDonough Sarcoma (FMS)-like tyrosine kinase 3 (FLT3)—particularly FLT3 internal tandem duplication (FLT3-ITD)—are among the most significant genetic alterations in AML. Patients with FLT3-ITD–positive AML face a heightened risk of relapse and historically poorer survival outcomes, underscoring the importance of targeted treatment strategies.

The integration of FLT3 inhibitors into frontline treatment strategies has reshaped the therapeutic landscape for FLT3-ITD–positive AML. Quizartinib is a highly selective, potent, type II FLT3 inhibitor that targets FLT3-ITD mutations, leading to inhibition of aberrant FLT3 signaling and suppression of leukemic cell proliferation. Clinical evidence supporting its use has expanded considerably, most notably through the QuANTUM-First trial, which evaluated quizartinib in combination with standard induction and consolidation chemotherapy followed by maintenance therapy in newly diagnosed FLT3-ITD–positive AML. The trial demonstrated a meaningful improvement in overall survival and extended duration of remission, reinforcing the value of incorporating targeted therapy into standard treatment regimens.

Beyond efficacy, optimizing patient outcomes requires careful attention to safety and practical implementation. Quizartinib is generally well tolerated; however, clinicians must monitor for treatment-emergent adverse events—such as QT interval prolongation, cytopenias, and infections—to ensure safe and sustained therapy. These events can be effectively managed through cardiac monitoring, dose adjustments, and supportive care.

This course features an expert interview with Professor Anskar Leung Yu-hung, a hematologist-oncologist who has published extensively on the pathogenesis and clinical management of AML. He discusses targeted therapy in newly diagnosed FLT3-ITD–positive AML, including clinical evidence from the QuANTUM-First trial. Learners will gain practical guidance on quizartinib use, safety considerations, and strategies to optimize patient outcomes, providing actionable insights to support evidence-based decision-making in real-world AML care.