title: Therapeutic options for patients with myelofibrosis in blast phase.
abstract: Myelofibrosis (MF) is a clonal stem cell disorder with the potential to transform to acute leukemia, referred to as myelofibrosis in blast phase (MF-BP). The outcome of patients with MF-BP is grave with a median survival of only 2.7 months. MF-BP is largely refractory to conventional chemotherapy and intensive induction therapy fails to have a significant impact with a median survival of 3.9 months. Eleven consecutive patients were treated at our institution with MF-BP over a 2-year period. Eligible patients with an available donor received an allogeneic stem cell transplant (ASCT) and those that were not eligible or without a donor were treated with Decitabine (DEC). The median time for follow up for the entire group was 9 months (range 5-21 month). At 9 months (range 5-45 months), 67% of the patients treated with DEC were alive and at 20 months (range 9-23 months), 53% of patients treated with ASCT remain alive. Reduced intensity conditioning allogeneic stem cell transplantation (RIC-ASCT) is a viable option that offers the potential for prolonged survival and the possibility of cure for patients with MF-BP. DEC is a tolerable outpatient chemotherapeutic regimen for MF-BP patients ineligible for transplant and deserves further prospective study.
authors.affiliation: Division of Hematology/Oncology, The Tisch Cancer Institute, New York, NY, USA. john.mascarenhas@mssm.edu
authors.forename: John
authors.initials: J
authors.lastname: Mascarenhas
authors.forename: Shyamala
authors.initials: S
authors.lastname: Navada
authors.forename: Adriana
authors.initials: A
authors.lastname: Malone
authors.forename: Amy
authors.initials: A
authors.lastname: Rodriguez
authors.forename: Vesna
authors.initials: V
authors.lastname: Najfeld
authors.forename: Ronald
authors.initials: R
authors.lastname: Hoffman
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D003131:Combined Modality Therapy; D006801:Humans; D055728:Primary Myelofibrosis / Q000628:therapy*; D033581:Stem Cell Transplantation
chemical_list: D000970:Antineoplastic Agents