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Ruxolitinib + Azacytidine in MDS/MPNs Evaluated

Am J Hematol; ePub 2017 Nov 14; Assi, et al

Ruxolitinib+azacytidine was well-tolerated in people with myelodysplastic syndrome/myeloproliferative neoplasms (MDS/MPNs), according to a phase 2 trial involving 35 individuals. Participants had unspecified MDS/MPNs (n=14), chronic myelomonocytic leukemia (CMML; n=17), or atypical chronic myeloid leukemia (aCML; n=4). They received ruxolitinib twice daily in 28-day cycles for the first 3 cycles. Azacytidine (between 25 and 75 mg/m2) IV or subcutaneously was added during the first 5 days of each subsequent cycle. It was given earlier or at higher dose when necessary. Among the results:

  • 57% of patients responded; 45% did so after azacytidine was added.
  • ~Two-thirds experienced a >50% reduction in palpable splenomegaly at 24 weeks.
  • More responders were JAK2-mutated and had splenomegaly.
  • 51% of responders experienced new onset grade 3/4 anemia and thrombocytopenia, vs 54% of nonresponders.
  • Patients with unspecified MDS/MPNs survived a median of 26.5 months.
  • In those with CMML and acML, survival was 15.1 and 8 months, respectively.


Assi R, Kantarjian H, Garcia-Manero G, et al. A phase II trial of ruxolitinib in combination with azacytidine in myelodysplastic syndrome/myeloproliferative neoplasms. [Published online ahead of print November 14, 2017]. Am J Hematol. doi:10.1002/ajh.24972.