MPN: The Molecular Drivers of Disease Initiation, Progression and Transformation and their Effect on Treatment

Cells. 2020 Aug 14;9(8):1901. doi: 10.3390/cells9081901.

Abstract

Myeloproliferative neoplasms (MPNs) constitute a group of disorders identified by an overproduction of cells derived from myeloid lineage. The majority of MPNs have an identifiable driver mutation responsible for cytokine-independent proliferative signalling. The acquisition of coexisting mutations in chromatin modifiers, spliceosome complex components, DNA methylation modifiers, tumour suppressors and transcriptional regulators have been identified as major pathways for disease progression and leukemic transformation. They also confer different sensitivities to therapeutic options. This review will explore the molecular basis of MPN pathogenesis and specifically examine the impact of coexisting mutations on disease biology and therapeutic options.

Keywords: CALR; DNA methylation; IFNα; JAK2; MPL; MPN; chromatin modifiers; driver mutations; leukemic transformation; myeloproliferation; spliceosome; transcriptional regulators; tumour suppressors.

Publication types

  • Review

MeSH terms

  • Animals
  • Bone Marrow Transplantation / methods*
  • Disease Progression*
  • Humans
  • Immune Checkpoint Inhibitors / therapeutic use*
  • Mice
  • Mutation*
  • Myeloproliferative Disorders / classification
  • Myeloproliferative Disorders / drug therapy*
  • Myeloproliferative Disorders / genetics*
  • Myeloproliferative Disorders / pathology
  • Phenotype
  • Prognosis
  • Transplantation, Homologous

Substances

  • Immune Checkpoint Inhibitors