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Molecular pathogenesis of atypical CML, CMML and MDS/MPN-unclassifiable

Int J Hematol. 2015 Mar;101(3):229-42. doi: 10.1007/s12185-014-1670-3. Epub 2014 Sep 12.

Abstract

According to the 2008 WHO classification, the category of myelodysplastic/myeloproliferative neoplasms (MDS/MPN) includes atypical chronic myeloid leukaemia (aCML), chronic myelomonocytic leukaemia (CMML), MDS/MPN-unclassifiable (MDS/MPN-U), juvenile myelomonocytic leukaemia (JMML) and a "provisional" entity, refractory anaemia with ring sideroblasts and thrombocytosis (RARS-T). The remarkable progress in our understanding of the somatic pathogenesis of MDS/MPN has made it clear that there is considerable overlap among these diseases at the molecular level, as well as layers of unexpected complexity. Deregulation of signalling plays an important role in many cases, and is clearly linked to more highly proliferative disease. Other mutations affect a range of other essential, interrelated cellular mechanisms, including epigenetic regulation, RNA splicing, transcription, and DNA damage response. The various combinations of mutations indicate a multi-step pathogenesis, which likely contributes to the marked clinical heterogeneity of these disorders. The delineation of complex clonal architectures may serve as the cornerstone for the identification of novel therapeutic targets and lead to better patient outcomes. This review summarizes some of the current knowledge of molecular pathogenetic lesions in the MDS/MPN subtypes that are seen in adults: atypical CML, CMML and MDS/MPN-U.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cytogenetic Analysis
  • DNA Damage
  • Epigenesis, Genetic
  • Gene Expression Regulation, Neoplastic
  • Humans
  • Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative / genetics*
  • Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative / metabolism
  • Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative / pathology
  • Leukemia, Myelomonocytic, Chronic / genetics*
  • Leukemia, Myelomonocytic, Chronic / metabolism
  • Leukemia, Myelomonocytic, Chronic / pathology
  • Mutation
  • Myelodysplastic-Myeloproliferative Diseases / classification
  • Myelodysplastic-Myeloproliferative Diseases / genetics*
  • Myelodysplastic-Myeloproliferative Diseases / metabolism
  • Myelodysplastic-Myeloproliferative Diseases / pathology
  • RNA Splicing
  • Signal Transduction