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.