Experimental therapy in myelofibrosis with myeloid metaplasia

Srdan Verstovsek, Alfonso Quintás-Cardama, Hagop Kantarjian, Ayalew Tefferi

Research output: Contribution to journalReview articlepeer-review

5 Scopus citations


Myelofibrosis with myeloid metaplasia (MMM) is a Philadelphia chromosome-negative myeloproliferative disorder that is characterised by constitutional symptoms, progressive anaemia and extramedullary haematopoiesis. There are no curative therapies available for patients with MMM apart from stem cell transplantation, which is associated with significant morbidity and mortality, and for which most patients are not suitable candidates. Traditional pharmacological therapy of MMM has focused on the palliation of symptoms associated with myeloproliferation and correction of cytopoenias. Recently, new findings regarding the molecular basis of MMM and the pathogenesis of the associated bone marrow stromal reaction have provided both basic and clinical researchers with invaluable tools to develop effective targeted therapies for patients with MMM. Several novel treatment strategies are being investigated including antiangiogenic agents, signal transduction inhibitors, inhibitors of fibrogenesis and small-molecule inhibitors of the JAK2V617F mutation. This article reviews the current status of experimental novel therapies for MMM.

Original languageEnglish (US)
Pages (from-to)1555-1563
Number of pages9
JournalExpert Opinion on Investigational Drugs
Issue number12
StatePublished - Dec 2006


  • Antiangiogenic agents
  • Antifibrotic agents
  • IMiDs
  • JAK2
  • Lenalidomide
  • Myelofibrosis
  • Pathogenesis
  • Tyrosine kinase inhibitors

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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