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Management of primary myelofibrosis

Author
Ayalew Tefferi, MD
Section Editor
Stanley L Schrier, MD
Deputy Editor
Rebecca F Connor, MD

INTRODUCTION

Primary myelofibrosis (PMF) is a myeloproliferative neoplasm characterized by the dysregulated proliferation of myeloid cells including megakaryocytes and myeloid and erythroid progenitors in the bone marrow resulting in ineffective erythropoiesis, the production of cytokines within the marrow microenvironment, and the reactive deposition of fibrous connective tissue (reticulin or collagen) in the bone marrow, often with osteosclerosis. In later fibrotic stages, the peripheral blood demonstrates teardrop-shaped red cells (ie, dacrocytes), nucleated red blood cells, and early myeloid forms (ie, a triad termed leukoerythroblastosis), and extramedullary hematopoiesis results in hepatomegaly and splenomegaly. PMF has had numerous names in the past, including agnogenic myeloid metaplasia, myelofibrosis with myeloid metaplasia, and chronic idiopathic myelofibrosis.

The related myeloproliferative neoplasms essential thrombocythemia (ET) and polycythemia vera (PV) can both undergo delayed disease transformation into a fibrotic state called post-ET myelofibrosis (post-ET MF) or post-PV MF, respectively. The conversion rates after 10 to 20 years of disease are less than 5 percent for ET and approximately 10 to 20 percent for PV. PMF, post-ET MF, and post-PV MF are all referred to as myelofibrosis.

The treatment of PMF will be reviewed here. An overview of the myeloproliferative neoplasms, as well as discussions of prognosis, pathogenetic mechanisms, clinical manifestations, and diagnosis of PMF, are presented separately. (See "Prognosis of primary myelofibrosis" and "Overview of the myeloproliferative neoplasms" and "Clinical manifestations and diagnosis of primary myelofibrosis" and "Pathogenetic mechanisms in primary myelofibrosis".)

GOALS OF CARE

At present, allogeneic hematopoietic cell transplantation (HCT) constitutes the only treatment modality with a curative potential in PMF. Other treatment modalities are, at best, palliative [1,2]. Most patients will not be candidates for HCT, and their care will focus on the alleviation of symptoms.

Most patients with PMF present with anemia, marked splenomegaly, early satiety, and hypercatabolic symptoms including severe fatigue, low-grade fever, night sweats, bone pain, and weight loss. During their clinical course, most patients experience massive splenomegaly, some hepatomegaly, along with progressive anemia requiring frequent red blood cell transfusions. Portal hypertension might accompany marked splenomegaly and could contribute to variceal bleeding or ascites. (See "Clinical manifestations and diagnosis of primary myelofibrosis".)

                                     

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Literature review current through: Jun 2016. | This topic last updated: Apr 13, 2016.
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