Primary myelofibrosis: Update on definition, pathogenesis, and treatment Journal Article


Authors: Abdel-Wahab, O. I.; Levine, R. L.
Article Title: Primary myelofibrosis: Update on definition, pathogenesis, and treatment
Abstract: Primary myelofibrosis (PMF) is a clonal stem cell disorder that manifests clinically as anemia, splenomegaly due to extramedullary hematopoiesis, leukoerythroblastosis, and constitutional symptoms, which are the clinical hallmarks of PMF.Within the past three years it has been determined that a single, recurrent, somatic mutation in the gene encoding the cytoplasmic tyrosine kinase Janus kinase 2 ( JAK2) occurs in the majority of patients with PMF, and more recently, activating mutations in the gene encoding the thrombopoietin receptor MPL have also been identified in a subset of PMF patients. These discoveries have yielded important insights into the pathogenesis of PMF and have brought about the first opportunity for rationally targeted therapy for this disorder. Here we present an updated review of the pathogenesis, definition, and treatment of PMF in light of the discovery of JAK2 and MPL mutations, as well as other recent work in the myeloproliferative neoplasm field. Copyright © 2009 by Annual Reviews. All rights reserved.
Keywords: treatment response; unclassified drug; gene mutation; chronic myelogenous leukemia (cml); essential thrombocythemia (et); extramedullary hematopoiesis (emh); janus kinase 2 (jak2); myeloproliferative neoplasm (mpn); polycythemia vera (pv); primary myelofibrosis (pmf); androgen; antianemic agent; at 9283; aurora kinase inhibitor; busulfan; cyclopropanecarboxylic acid [4 [4 (4 methyl 1 piperazinyl) 6 (5 methyl 2h pyrazol 3 ylamino) 2 pyrimidinylthio]phenyl]amide; danazol; fludarabine; histone deacetylase inhibitor; hydroxyurea; incb 01824; itf 2357; janus kinase 2; janus kinase inhibitor; lenalidomide; lestaurtinib; prednisone; tg 101209; tg 101348; thalidomide; thrombopoietin receptor; xl 019; allogeneic stem cell transplantation; clinical feature; clinical trial; constipation; disease course; drowsiness; drug activity; drug tolerability; fatigue; histopathology; mortality; myeloid metaplasia; neutropenia; paresthesia; pathogenesis; review; splenectomy; primary myelofibrosis
Journal Title: Annual Review of Medicine
Volume: 60
ISSN: 0066-4219
Publisher: Annual Reviews  
Date Published: 2009-01-01
Start Page: 233
End Page: 245
Language: English
DOI: 10.1146/annurev.med.60.041707.160528
PUBMED: 18947294
PROVIDER: scopus
DOI/URL:
Notes: --- - "Cited By (since 1996): 3" - "Export Date: 30 November 2010" - "CODEN: ARMCA" - "Source: Scopus"
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  1. Ross Levine
    782 Levine