Therapy with the histone deacetylase inhibitor pracinostat for patients with myelofibrosis

Alfonso Quintás-Cardama, Hagop Kantarjian, Zeev Estrov, Gautam Borthakur, Jorge Cortes, Srdan Verstovsek

Research output: Contribution to journalArticlepeer-review

66 Scopus citations


Approximately half of the patients with myelofibrosis (MF) carry mutant JAK2 V617F proteins. JAK2 V617F has been recently shown to translocate to the nucleus and modify specific histones, thus regulating transcription. We report on a phase II study testing the activity and tolerability of the histone deacetylase inhibitor pracinostat given at 60mg every other day for three weeks per month in 22 patients with intermediate or high risk MF. Eight (36%) patients experienced clinical benefit, with 6 (27%) experiencing reductions in splenomegaly (median 3cm, range 1-4cm). According to International Working Group criteria, 2 (9%) patients had clinical improvement (anemia response in both cases). The most frequent side effect associated to pracinostat therapy was fatigue, which occurred in 20 (91%) patients (grade 2 in 3 patients). Grade 3-4 neutropenia, anemia, and thrombocytopenia occurred in 13%, 0%, and 21%, respectively. Twenty-one patients permanently discontinued pracinostat, mainly due to lack of efficacy. In conclusion, pracinostat at the dose tested is reasonably tolerated and has modest activity in patients with MF.

Original languageEnglish (US)
Pages (from-to)1124-1127
Number of pages4
JournalLeukemia Research
Issue number9
StatePublished - Sep 2012
Externally publishedYes


  • Histone deacetylase inhibitor
  • JAK2
  • Myelofibrosis
  • Pracinostat
  • SB939

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


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