Low-dose ruxolitinib shows effective in treating myelofibrosis

Ann Hematol. 2021 Jan;100(1):135-141. doi: 10.1007/s00277-020-04311-z. Epub 2020 Oct 20.

Abstract

The aim of this study was to investigate the effect of low-dose ruxolitinib (daily dose ≤ 10 mg) for the treatment of myelofibrosis (MF). A retrospective analysis was performed on a total of 88 patients with myeloproliferative neoplasm-associated MF (MPN-MF) who were diagnosed and treated in West China Hospital, Sichuan University, China. A total of 44 MPN-MF patients received a low dose of ruxolitinib (daily dose ≤ 10 mg), while another 44 patients received 10-25 mg twice daily. Low-dose ruxolitinib treatment resulted in slow, but gradual spleen response. Compared with baseline, the mean changes in palpable spleen length in the low- and high-dose groups were -26.9 and -49.0% after 12 weeks of treatment, respectively, and -46.7 and -64.1% after 48 weeks of treatment, respectively. In the low dose group, the median myeloproliferative neoplasm symptom assessment form (MPN-SAF) total symptom score (TSS) decreased by 37.8 and 35.9% at the 12 weeks and 48 weeks after treatment, respectively. No statistical difference was observed in MPN-SAF TSS among different dose groups. After 48 weeks of treatment, bone marrow (BM) fibrosis improved in 43.3% (13/30) of evaluated patients and was stable in 56.7% (17/30) patients. In the low-dose treated group, BM fibrosis improved in 50% patients and was stable in remaining 50%. Low-dose ruxolitinib is effective in treating MF.

Keywords: Bone marrow fibrosis; Dose; Myelofibrosis; Ruxolitinib; Splenomegaly.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Dose-Response Relationship, Drug
  • Female
  • Humans
  • Janus Kinase Inhibitors / administration & dosage*
  • Male
  • Middle Aged
  • Nitriles
  • Primary Myelofibrosis / drug therapy*
  • Primary Myelofibrosis / pathology
  • Pyrazoles / administration & dosage*
  • Pyrimidines
  • Retrospective Studies
  • Spleen / drug effects*
  • Spleen / pathology
  • Treatment Outcome

Substances

  • Janus Kinase Inhibitors
  • Nitriles
  • Pyrazoles
  • Pyrimidines
  • ruxolitinib