Conventional and experimental drug therapy in myelofibrosis with myeloid metaplasia |
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Authors: | Ruben A Mesa MD Alfonso Quintás-Cardama MD Srdan Verstovsek MD PhD |
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Institution: | (1) Division of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA |
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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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