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Conventional and experimental drug therapy in myelofibrosis with myeloid metaplasia

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Abstract

Myelofibrosis with myeloid metaplasia (MMM) is currently classified as a classic (ie, BCR-ABL-negative) myeloproliferative disorder characterized by anemia, multiorgan extramedullary hematopoiesis, constitutional symptoms, and premature death from either leukemic transformation or other disease complications. Stem cell transplantation can be curative, but many patients either are not appropriate candidates or do not choose to accept the significant risks associated with transplantation. Current pharmacologic therapy has been beneficial mainly in terms of palliating disease-associated cytopenias, constitutional symptoms, splenomegaly, and other organ damage from excess myeloproliferation. Novel treatment strategies are under investigation, including targeted inhibition of JAK2V617F, the activating tyrosine kinase point mutation present in about half of patients with MMM. In this article, we review both the old and new pharmacologic options for MMM.

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  1. Division of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN, 55905, USA

    Ruben A. Mesa MD

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  1. Ruben A. Mesa MD
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  2. Alfonso Quintás-Cardama MD
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  3. Srdan Verstovsek MD, PhD
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Correspondence to Ruben A. Mesa MD.

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Mesa, R.A., Quintás-Cardama, A. & Verstovsek, S. Conventional and experimental drug therapy in myelofibrosis with myeloid metaplasia. Curr Hematol Malig Rep 2, 25–33 (2007). https://doi.org/10.1007/s11899-007-0004-4

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