Blastic Transformation of Classic Myeloproliferative Neoplasms

  • Ruben A. MesaEmail author
Part of the Contemporary Hematology book series (CH)


The BCR-ABL negative myeloproliferative neoplasms (MPNs) of essential thrombocythemia, polycythemia vera, and primary myelofibrosis have an increasing predisposition over the course of a patient’s illness to transform to overt acute leukemia what is referred to as MPN-Blast Phase (MPN-BP). Current therapies for the MPNs are limited, and no therapy other than allogeneic stem cell transplant has clearly altered the natural history of these neoplasms. Pathogenetic mechanisms which lead to an MPN progressing to MPN-BP are incompletely understood, but seem to correlate with accumulation of additional karyotypic abnormalities as opposed to increases in MPN-associated molecular lesion burden (such as JAK2V617F). The development of MPN-BP is heralded by worsening cytopenias, constitutional symptoms, and a very poor survival despite therapeutic intervention. Risk factors for developing MPN-BP include both features at diagnosis (such as increased peripheral blood blasts, karyotypic abnormalities, and thrombocytopenia), as well as exposure to established agents which enhance leukemogenesis (i.e., P-32 and alkylators). Salvage of MPN-BP by induction chemotherapy followed by allogeneic stem cell transplant is possible, but a successful option in a small minority of patients. Multiple avenues of therapeutic investigation are ongoing to treat, or prevent, MPN-BP including early allogeneic stem cell transplantation, hypomethylating agents, and JAK2 inhibition. An improved understanding of the pathogenetic underpinings of MPN-BP are necessary if more effective targeted therapies are to be developed.


Myeloproliferative disorder Blast phase Acute myeloid leukemia Leukemic transformation 


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© Springer Science+Business Media, LLC 2011

Authors and Affiliations

  1. 1.Consultant of Hematology & OncologyMayo ClinicScottsdaleUSA

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