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A Phase I Study of the Mitochondrial Metabolism Inhibitor CPI-613 for AML

Timothy S. Pardee, MD
Published Online:7:41 PM, Mon June 23, 2014
Timothy S. Pardee, MD, assistant professor, Hematology & Oncology, Wake Forest University Baptist Medical Center, discusses the results of a phase I study of the mitochondrial metabolism inhibitor CPI-613 for relapsed or refractory acute myeloid leukemia (AML).

Clinical Pearls

  • 24 patients with relapsed or refractory AML received the mitochondrial metabolism inhibitor CPI-613 in combination with high-dose cytarabine and mitoxantrone.
  • The response rate was 54% (11 complete remissions + 2 complete remissions with incomplete count recovery).
  • Patients with poor risk cytogenetics tend to respond poorly to chemotherapy.
  • In this trial, the response rate of patients with poor risk cytogenetics was 53% (7/13).
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