Therapeutic benefit of decitabine, a hypomethylating agent, in patients with high-risk primary myelofibrosis and myeloproliferative neoplasm in accelerated or blastic/acute myeloid leukemia phase

Talha Badar, Hagop M. Kantarjian, Farhad Ravandi, Elias Jabbour, Gautam Borthakur, Jorge E. Cortes, Naveen Pemmaraju, Sherry R. Pierce, Kate J. Newberry, Naval Daver, Srdan Verstovsek

Research output: Contribution to journalArticlepeer-review

67 Scopus citations

Abstract

Myeloproliferative neoplasm (MPN) transformed to acute myeloid leukemia (MPN-AML), MPN in accelerated phase (MPN-AP), and high-risk primary myelofibrosis (PMF) are associated with a poor response to therapy and very short survival. Several reports have suggested clinical activity of hypomethylating agents in these patients. We conducted a retrospective study of 21 patients with MPN-AML, 13 with MPN-AP and 11 with DIPSS-plus high-risk PMF treated with decitabine at our institution over the last 7 years and evaluated their clinical outcomes. Six patients (29%) with MPN-AML responded to decitabine (3 CR, 2 CRi, and 1 PR); median response duration was 7 months. The median overall survival (OS) was significantly higher in those who responded (10.5 vs 4 months). Among patients with MPN-AP, 8 patients (62%) benefited; the median response duration was 6.5 months. The median OS was 11.8 months in responders vs 4.7 months in non-responders. Among patients with DIPSS-plus high-risk PMF, 9 (82%) benefited; the median response duration was 9 months. The median OS was 32 months in responders vs 16.3 months in non-responders. Decitabine is a viable therapeutic option for patients with MPN-AML, MP-AP and high-risk PMF. Prospective clinical studies combining decitabine with other clinically active agents are needed to improve overall outcome.

Original languageEnglish (US)
Pages (from-to)950-956
Number of pages7
JournalLeukemia Research
Volume39
Issue number9
DOIs
StatePublished - Sep 1 2015
Externally publishedYes

Keywords

  • AML
  • Accelerated phase
  • Decitabine
  • Myelofibrosis
  • Myeloproliferative neoplasm

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

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