A Phase II Trial of Imatinib Mesylate as Maintenance Therapy for Patients With Newly Diagnosed C-kit–positive Acute Myeloid Leukemia

Anjali S. Advani, William Tse, Hong Li, Xuefei Jia, Paul Elson, Brenda Cooper, Francis Ali-Osman, Jino Park, Arati V. Rao, David A. Rizzieri, Eunice S. Wang, Claudiu V. Cotta, Matt Kalaycio, Ronald M. Sobecks, Basel Rouphail, Jaroslaw P. Maciejewski, Jaime Fensterl, Jennifer S. Carew, Bethany Foster, Mary Lynn RushBarbara Tripp, Donna Adams, Donna Corrigan, Elizabeth A. Griffiths, Mikkael A. Sekeres

Research output: Contribution to journalArticlepeer-review

3 Scopus citations


Introduction: Adults with acute myeloid leukemia (AML) have a high rate of remission; however, more than 50% relapse. C-kit is expressed in approximately 60% of patients with de novo AML 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 (defined as no relapse and no death) compared with historical controls. Results: The median progression-free survival of patients < 60 years of age was 52.1 months (historical control, 13 months) and for 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. Conclusions: Imatinib maintenance therapy may improve the outcome of newly diagnosed patients with AML who are < 60 years of age. The prognosis of acute myeloid leukemia remains poor, and novel treatments are needed. C-kit is expressed on the surface of acute myeloid leukemia cells and represents a potential target. This trial examined the addition of the c-kit inhibitor, imatinib, for 12 months after the completion of chemotherapy. The addition of imatinib appeared to improve outcomes in patients < 60 years of age.

Original languageEnglish (US)
Pages (from-to)113-118
Number of pages6
JournalClinical Lymphoma, Myeloma and Leukemia
Issue number2
StatePublished - Feb 2021
Externally publishedYes


  • AF1q
  • CD117
  • Prognosis
  • Survival
  • Targeted

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


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