The Treatment Landscape of Myelofibrosis Before and After Ruxolitinib Approval

Clin Lymphoma Myeloma Leuk. 2017 Dec;17(12):e45-e53. doi: 10.1016/j.clml.2017.08.002. Epub 2017 Aug 5.


Introduction/background: Myelofibrosis (MF) is a chronic myeloproliferative neoplasm that presents with a heterogeneous clinical phenotype and prognosis. Before the US Food and Drug Administration approval of ruxolitinib, treatment options were varied and had limited effect. The increased use of ruxolitinib has drastically altered the MF treatment landscape. In this study, we aimed to clarify the clinical situations in which ruxolitinib is being used and analyze its effect on this landscape.

Patients and methods: We retrospectively assessed treatment choices for MF patients treated at our institution (n = 309). This population was divided into 2 cohorts on the basis of a diagnosis before (cohort BR: n = 174) or after (cohort AR: n = 135) ruxolitinib approval. Cohorts were further stratified for comparison according to presenting clinical factors.

Results: Expectedly, the first-line use of ruxolitinib markedly increased after its approval. AR patients were less likely to receive erythropoiesis-stimulating agents (ESAs; P = .0003) and thalidomide (P = .003) than BR patients. In patients with MF-related symptoms and/or splenomegaly, increased use of ruxolitinib was associated with decreased use of first-line ESA (P = .03) or thalidomide (P = .03). In anemic patients, increased use of first-line ruxolitinib was associated with a decreased use of thalidomide (P = .007). In patients with severe leukocytosis, ruxolitinib use did not significantly increase and hydroxyurea was the preferred first-line agent.

Conclusion: Overall, the increased use of ruxolitinib appears to have come predominantly at the expense of thalidomide and ESAs, while not having a large effect on the first-line use of hydroxyurea.

Keywords: Hydroxyurea; JAK2 inhibitor; Myeloproliferative neoplasm; Thalidomide; Treatment.

MeSH terms

  • Aged
  • Drug Approval*
  • Drug Therapy / statistics & numerical data
  • Drug Therapy / trends
  • Female
  • Hematinics / therapeutic use*
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Male
  • Nitriles
  • Primary Myelofibrosis / drug therapy*
  • Pyrazoles / therapeutic use*
  • Pyrimidines
  • Retrospective Studies
  • Thalidomide / therapeutic use*


  • Hematinics
  • Immunosuppressive Agents
  • Nitriles
  • Pyrazoles
  • Pyrimidines
  • Thalidomide
  • ruxolitinib