Omacetaxine mepesuccinate in patients with advanced chronic myeloid leukemia with resistance or intolerance to tyrosine kinase inhibitors

H. Jean Khoury, Jorge Cortes, Michele Baccarani, Meir Wetzler, Tamas Masszi, Raghunadharao Digumarti, Adam Craig, Annie Claude Benichou, Luke Akard

Research output: Article

14 Citations (Scopus)


Omacetaxine mepesuccinate promotes apoptosis by inhibiting the production of short-lived oncoproteins. The efficacy and safety of omacetaxine in patients with advanced chronic myeloid leukemia (CML) previously treated with tyrosine kinase inhibitors were assessed in two phase II trials (CML-202 and CML-203). Fifty-one patients in accelerated phase (AP-CML) and 44 in myeloid blast phase (BP-CML) received subcutaneous omacetaxine 1.25 mg/m2 twice daily days 1-14 every 28 days until hematologic response/improvement or any cytogenetic response, then days 1-7 every 28 days until disease progression. The primary endpoint was maintenance or attainment of a major hematologic response (MHR). Cytogenetic responses were also evaluated. MHR was 37% in patients with AP-CML and 9% with BP-CML (22% and 5% in those with a history of T315I). Most grade 3/4 adverse events were related to myelosuppression, and were generally manageable. Omacetaxine demonstrates activity and an acceptable safety profile in pretreated patients with advanced CML, irrespective of mutational status.

Original languageEnglish
Pages (from-to)120-127
Number of pages8
JournalLeukemia and Lymphoma
Issue number1
Publication statusPublished - jan. 1 2015


ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

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