Anemia in myelofibrosis: Current and emerging treatment options

Crit Rev Oncol Hematol. 2022 Dec:180:103862. doi: 10.1016/j.critrevonc.2022.103862. Epub 2022 Nov 1.

Abstract

Myelofibrosis (MF) is a clonal hematologic malignancy with progressive bone marrow fibrosis. Clinical manifestations of MF include splenomegaly, constitutional symptoms, and anemia, whose pathogenesis is multifactorial and largely due to ineffective erythropoiesis and is clinically associated with poor quality of life and reduced overall survival. The only curative treatment for MF is allogenic stem cell transplantation; however, few patients are eligible. Disease management strategies for MF-related anemia have limited effectiveness, and Janus kinase (JAK) inhibitors may induce or worsen related anemia. Thus, there is a significant unmet need for the treatment of patients with MF-related anemia. This review summarizes current and emerging treatments for anemia in MF, including luspatercept and KER-050 (transforming growth factor-β ligand traps), momelotinib and pacritinib (JAK inhibitors), pelabresib (a bromodomain extra-terminal domain inhibitor), PRM-151 (an antifibrotic agent), imetelstat (a telomerase inhibitor), and navitoclax (a BCL-2/BCL-xL inhibitor). Therapeutic combinations with ruxolitinib may offer another treatment approach.

Keywords: Anemia; Myelofibrosis.

Publication types

  • Review

MeSH terms

  • Anemia* / drug therapy
  • Anemia* / therapy
  • Antineoplastic Agents* / therapeutic use
  • Humans
  • Janus Kinase 2
  • Nitriles / therapeutic use
  • Primary Myelofibrosis* / complications
  • Primary Myelofibrosis* / therapy
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use
  • Quality of Life

Substances

  • Janus Kinase 2
  • Nitriles
  • Antineoplastic Agents
  • Protein Kinase Inhibitors