Management of myeloproliferative neoplasms: from academic guidelines to clinical practice

Curr Hematol Malig Rep. 2012 Mar;7(1):50-6. doi: 10.1007/s11899-011-0109-7.

Abstract

Myeloproliferative neoplasms (MPNs) are clonal disorders characterized by excessive production of mature cells. In most of the classic Philadelphia-negative MPNs-polycythemia vera (PV), essential thrombocythemia (ET), and MPN-associated myelofibrosis (MPN-MF)-oncogenic mutations affecting JAK2 or MPL lead to constitutive activation of cytokine-regulated intracellular signalling pathways. The traditional therapy for PV and ET is the prevention of thrombotic events with antiproliferative agents in association with aspirin. New drugs such as pegylated interferon and anti-JAK agents are candidates for slowing the evolution to myelofibrosis or leukemia. Conventional therapy for MPN-MF is driven by clinical needs, primarily anemia and splenomegaly. Lenalidomide and pomalidomide are new candidates for treating anemia. JAK2 ATP-competitive inhibitors or drugs that indirectly inhibit the JAK-STAT pathway, like RAD001, are the new candidates for splenomegaly in MPN-MF, but in spite of their strong rationale, they have shown only a palliative benefit. Allogeneic stem cell transplantation remains the only potentially curative approach.

Publication types

  • Review

MeSH terms

  • Anemia / prevention & control
  • Anti-Inflammatory Agents, Non-Steroidal / therapeutic use
  • Antineoplastic Agents / therapeutic use*
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Janus Kinase 2 / antagonists & inhibitors
  • Molecular Targeted Therapy / methods
  • Myeloproliferative Disorders / complications
  • Myeloproliferative Disorders / genetics
  • Myeloproliferative Disorders / therapy*
  • Nitriles
  • Polycythemia Vera / genetics
  • Polycythemia Vera / therapy
  • Practice Guidelines as Topic
  • Protein Kinase Inhibitors / therapeutic use*
  • Pyrazoles / therapeutic use
  • Pyrimidines
  • Risk Assessment
  • Thrombocythemia, Essential / complications
  • Thrombocythemia, Essential / genetics
  • Thrombocythemia, Essential / therapy
  • Venous Thrombosis / prevention & control

Substances

  • Anti-Inflammatory Agents, Non-Steroidal
  • Antineoplastic Agents
  • Nitriles
  • Protein Kinase Inhibitors
  • Pyrazoles
  • Pyrimidines
  • ruxolitinib
  • Janus Kinase 2