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Diagnosis and Management of Prefibrotic Primary Myelofibrosis (Pre-PMF)

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Pathogenesis and Treatment of Leukemia

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Abstract

This review is focused on prefibrotic primary myelofibrosis (pre-PMF) and evaluates clinical impact in relation to World Health Organization-defined essential thrombocythemia (ET). Compared to ET, patients with pre-PMF present with higher leukocyte counts, lower hemoglobin levels, higher lactate dehydrogenase values, and more frequently palpable splenomegaly. Incidences of JAK2V617F and CALR-mutated pre-PMF patients are superimposable to ET. Nondriver high-risk mutations in ASXL1, EZH2, SRSF2, and IDH 1/2 are increased in pre-PMF compared to ET. Vascular complications are not significantly different while hemorrhagic events are increased in pre-PMF. Median survival (around 13 vs. 19 years), 10-year cumulative rates progression to overt myelofibrosis, and transformation to blast phase are significantly different for pre-PMF versus ET. Primary objective of therapy is to prevent major thrombohemorrhagic complications. Because most pre-PMF patients fall in the lower prognostic IPSS group, observation alone or aspirin is recommended, while patients at intermediate risk may require a symptom-driven treatment and high-risk patients need cytostatic drugs.

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Authors and Affiliations

  1. FROM Research Foundation, Papa Giovanni XXIII Hospital, Bergamo, Italy

    Tiziano Barbui & Alessandra Carobbio

  2. Institute of Pathology, University of Cologne, Cologne, Germany

    Jürgen Thiele

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  1. Tiziano Barbui
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  2. Alessandra Carobbio
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  3. Jürgen Thiele
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Correspondence to Tiziano Barbui .

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  1. Department of Medicine, Queen Mary Hospital, Hongkong, Hong Kong

    Harinder Gill

  2. Department of Medicine, Queen Mary Hospital, Hongkong, Hong Kong

    Yok-Lam Kwong

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Barbui, T., Carobbio, A., Thiele, J. (2023). Diagnosis and Management of Prefibrotic Primary Myelofibrosis (Pre-PMF). In: Gill, H., Kwong, YL. (eds) Pathogenesis and Treatment of Leukemia. Springer, Singapore. https://doi.org/10.1007/978-981-99-3810-0_41

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