Leukemic transformation in myeloproliferative neoplasms: therapy-related or unrelated?

Best Pract Res Clin Haematol. 2014 Jun;27(2):141-53. doi: 10.1016/j.beha.2014.07.003. Epub 2014 Jul 19.

Abstract

Polycythemia vera, essential thrombocythemia, and primary myleofibrosis are chronic myeloproliferative neoplasms (MPNs) associated with an increased morbidity and mortality. MPNs are also associated with progression to acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The "true" rate of transformation is not known mainly due to selection bias in clinical trials and underreporting in population-based studies. The outcome after transformation is dismal. The underlying mechanisms of transformation are incompletely understood and in part remain an area of controversy. There is an intrinsic propensity in MPNs to progress to AML/MDS, the magnitude of which is not fully known, supporting a role for nontreatment-related factors. High doses of alkylating agents, P(32) and combined cytoreductive treatments undoubtedly increase the risk of transformation. The potential leukemogenic role of hydroxyurea has been a matter of debate due to difficulties in performing large prospective randomized trials addressing this issue. The main focus of this review is to elucidate therapy-related leukemic transformation in MPNs with a special focus on the role of hydroxyurea.

Keywords: JAK2 mutation; X-ray treatment; alkylators; antineoplastic agents; essential thrombocythemia; hydroxyurea; leukemia etiology; leukemic transformation; myeloproliferative neoplasm; polycythemia vera; primary myelofibrosis; radioactive phosphorous; risk factor.

Publication types

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

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Cell Transformation, Neoplastic / drug effects
  • Cell Transformation, Neoplastic / pathology
  • Disease Progression
  • Humans
  • Hydroxyurea / therapeutic use
  • Janus Kinase 2 / genetics
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / etiology
  • Leukemia, Myeloid, Acute / genetics
  • Leukemia, Myeloid, Acute / mortality
  • Mutation
  • Myelodysplastic Syndromes / drug therapy*
  • Myelodysplastic Syndromes / etiology
  • Myelodysplastic Syndromes / genetics
  • Myelodysplastic Syndromes / mortality
  • Phosphorus Radioisotopes / therapeutic use
  • Polycythemia Vera / complications
  • Polycythemia Vera / drug therapy*
  • Polycythemia Vera / genetics
  • Polycythemia Vera / mortality
  • Primary Myelofibrosis / complications
  • Primary Myelofibrosis / drug therapy*
  • Primary Myelofibrosis / genetics
  • Primary Myelofibrosis / mortality
  • Survival Analysis
  • Thrombocythemia, Essential / complications
  • Thrombocythemia, Essential / drug therapy*
  • Thrombocythemia, Essential / genetics
  • Thrombocythemia, Essential / mortality

Substances

  • Antineoplastic Agents
  • Phosphorus Radioisotopes
  • JAK2 protein, human
  • Janus Kinase 2
  • Hydroxyurea