Novel strategies for patients with chronic myeloproliterative disorders

Giovanni Barosi, Vittorio Rosti

Research output: Contribution to journalArticle


Purpose of review This review focuses on new strategies for unmet clinical needs and on new targeted therapies in classical Philadelphia-negative myeloproliferative neoplasms. Recent findings Meta-analyses in essential thrombocythemia documented Janus kinase 2 (JAK2) V617F as being associated with increased risk of thrombosis. New studies reinforced the evidence of leukocytosis as an independent risk factor for thrombosis in polycythemia vera and essential thrombocythemia. In a phase II trial of pegylated interferon-alpha2a in polycythemia vera patients, a decrease of JAK2 mutant expression to undetectable levels was demonstrated. New trials documented that 5-azacytidine and bortezomib have negligible effect in primary myelofibrosis, whereas thalidomide and tipifarnib produce 22 and 44% response, respectively. In primary myelofibrosis, the JAK2 inhibitor, INCB018424, resulted in a rapid and marked reduction in splenomegaly and a clinical improvement, with a modest effect on JAK2 V617F burden. Summary Treating low-risk essential thrombocythemia and polycythemia vera patients presenting with leukocytosis or JAK2 V617F mutation in order to prevent thrombosis deserves a prospective validation. Pursuing clonal remission in polycythemia vera by interferon needs new evidence. Tipifarnib may be added to conventional therapeutic instruments for symptomatic primary myelofibrosis. The results of anti-JAK2-targeted therapies are encouraging as regards symptoms reduction but not clonal remission.

Original languageEnglish
Pages (from-to)129-134
Number of pages6
JournalCurrent Opinion in Hematology
Issue number2
Publication statusPublished - Mar 2009



  • Chronic myeloproliferative neoplasm
  • Essential thrombocythemia
  • Janus kinase 2 V617F
  • Polycythemia vera
  • Primary myelofibrosis

ASJC Scopus subject areas

  • Hematology

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