Prognosis of primary myelofibrosis
- Ayalew Tefferi, MD
Ayalew Tefferi, MD
- Professor of Medicine and Hematology
- Mayo Medical School
Primary myelofibrosis (PMF) is a myeloproliferative neoplasm characterized by the dysregulated proliferation of megakaryocytes and granulocytes 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 (MF).
The prognosis of PMF will be reviewed here. An overview of the myeloproliferative neoplasms, as well as discussions of treatment, pathogenetic mechanisms, clinical manifestations, and diagnosis of PMF, are presented separately. (See "Overview of the myeloproliferative neoplasms" and "Clinical manifestations and diagnosis of primary myelofibrosis" and "Management of primary myelofibrosis" and "Pathogenetic mechanisms in primary myelofibrosis".)
In an epidemiologic study of patients within Olmsted County, Minnesota, the three-year survival rate was 52 percent . Longer survival times have been reported in several non-population-based studies, including the series used for constructing the International Prognostic Scoring System (IPSS) for PMF, in which the median survival was reported to be 69 months .
A number of prognostic models are available for assessing prognosis in PMF [2-10]. The most recent and internationally recognized is the DIPSS Plus for PMF, described below. Their use in risk stratification for treatment decisions is presented separately. (See "Management of primary myelofibrosis".)
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- International Working Group Scoring System
- - IPSS
- - Dynamic IPSS
- - DIPSS Plus
- Predicting leukemic transformation
- - Elevated cytokine levels
- - JAK2, calreticulin, and MPL mutational burden
- - Effect of other mutations
- Candidates for hematopoietic cell transplantation
- RISK STRATIFICATION FOR MANAGEMENT
- SUMMARY AND RECOMMENDATIONS