A Phase 2 Trial of Imatinib Mesylate as Maintenance Therapy for Patients with Newly Diagnosed C-kit Positive Acute Myeloid Leukemia

Abstract: Introduction Adults with AML have a high rate of remission; however, more than 50% relapse. C-kit is expressed in approximately 60% of de novo AML patients and represents a potential therapeutic target. Materials and Methods: Patients with newly diagnosed AML received 12 months of imatinib mesylate as maintenance therapy after the completion of post-remission therapy. The primary objective was to determine whether this approach improved progression-free survival (PFS) (defined as no relapse and no death) compared to historical controls. Results: The median PFS of patients > 60 years of age was 10.7 months (historical control: 8 months). The median level of AF1q expression was high (9.59) and 84% of patients had moderate or high levels of drug-resistance factors. Conclusion: Imatinib maintenance therapy may improve the outcome of newly diagnosed patients with AML