Original Article

Annals of Hematology

, Volume 91, Issue 1, pp 33-38

First online:

Phase II study of the histone deacetylase inhibitor belinostat (PXD101) for the treatment of myelodysplastic syndrome (MDS)

  • Amanda CashenAffiliated withWashington University School of Medicine Email author 
  • , Mark JuckettAffiliated withUniversity of Wisconsin
  • , Alcee JumonvilleAffiliated withGundersen Clinic, Ltd.
  • , Mark LitzowAffiliated withMayo Clinic Rochester
  • , P. J. FlynnAffiliated withMinnesota Oncology Hematology
  • , John EckardtAffiliated withCenter for Cancer Care and Research
  • , Betsy LaPlantAffiliated withMayo Clinic Cancer Center
  • , Kristina LaumannAffiliated withMayo Clinic Cancer Center
  • , Charles ErlichmanAffiliated withMayo Clinic Cancer Center
    • , John DiPersioAffiliated withWashington University School of Medicine

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The inhibition of histone deacetylase (HDAC) can induce differentiation, growth arrest, and apoptosis in cancer cells. This phase II multicenter study was undertaken to estimate the efficacy of belinostat, a potent inhibitor of both class I and class II HDAC enzymes, for the treatment of myelodysplastic syndrome (MDS). Adults with MDS and ≤2 prior therapies were treated with belinostat 1,000 mg/m2 IV on days 1–5 of a 21-day cycle. The primary endpoint was a proportion of confirmed responses during the first 12 weeks of treatment. Responding patients could receive additional cycles until disease progression or unacceptable toxicity. Twenty-one patients were enrolled, and all were evaluable. Patients were a median 13.4 months from diagnosis, and 14 patients (67%) had less than 5% bone marrow blasts. Seventeen patients (81%) were transfusion dependent. Prior therapy included azacytidine (n = 7) and chemotherapy (n = 8). The patients were treated with a median of four cycles (range, 1–8) of belinostat. There was one confirmed response—hematologic improvement in neutrophils—for an overall response rate of 5% (95% CI, 0.2–23). Median overall survival was 17.9 months. Grades 3–4 toxicities considered at least to be possibly related to belinostat were: neutropenia (n = 10), thrombocytopenia (n = 9), anemia (n = 5), fatigue (n = 2), febrile neutropenia (n = 1), headache (n = 1), and QTc prolongation (n = 1). Because the study met the stopping rule in the first stage of enrollment, it was closed to further accrual.


Belinostat PXD101 Myelodysplastic syndrome Histone deacetylase inhibitor