Oncogenic Signals as Treatment Targets in Classic Myeloproliferative Neoplasms

Ayalew Tefferi, Ross L. Levine, Hagop Kantarjian

Research output: Contribution to journalArticlepeer-review

5 Scopus citations


Therapeutically validated oncoproteins in myeloproliferative neoplasms (MPNs) include BCR-ABL in chronic myelogenous leukemia (CML) and a spectrum of PDGFRA/B mutant proteins that are products of intra- (eg, FIP1L1-PDGFRA) or interchromosomal (eg, ETV6-PDGFRB) gene fusions. Other MPN-relevant putative oncogenes that are awaiting therapeutic validation, include JAK2 and MPL mutations in polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF); KITD816V and other KIT mutations in systemic mastocytosis, and FGFR1 rearrangements associated with the 8p11 leukemia/lymphoma syndrome. The current review focuses on mutant molecules of interest in classic MPNs (ie, CML, PV, ET, and PMF) in the context of their value as drug targets.

Original languageEnglish (US)
Pages (from-to)114-119
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Issue number1 SUPPL.
StatePublished - Jan 2009


  • Polycythemia
  • leukemia
  • mutation
  • myelofibrosis
  • thrombocythemia

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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