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Primary myelofibrosis and its targeted therapy

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Abstract

Primary myelofibrosis is a unique entity among BCR-ABL-negative myeloproliferative diseases, manifesting as bone marrow fibrosis and pancytopenia. Considerable evidence indicates that genetic and epigenetic abnormalities can result in defective clonal hematopoietic stem cell proliferation in addition to bone marrow microenvironment alteration. The “bad seeds in bad soil” theory illustrates the orchestrating efforts of hematopoietic stem cells, stromal cells, and their surrounding signaling molecules in myelofibrosis progression and malignancy transformation, though the exact mechanism of myelofibrosis is still not clear. This study reviews current concepts and questions regarding the pathogenesis of primary myelofibrosis and discusses the emerging targeted therapy aimed at restoring normal bone marrow environment and halting bone marrow fibrotic deterioration.

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Acknowledgments

The authors thank the patients and their family for contributing invaluable knowledge and experience for this study and also would like to thank Dr. Jerry Spivak for providing sincere comments on this manuscript. This study was supported by: AA&MDSIF research grant to JJP (146818), American Cancer Society grant to JJP (124171-IRG-13-043-02), and A Pennsylvania State University College of Medicine research grant to JJP.

Authorship

Contribution: LS, KB, and JJP participated in manuscript writing. JJP designed this study.

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Authors and Affiliations

  1. Department of Medicine, Duke University School of Medicine, Durham, NC, USA

    Lindsey Shantzer

  2. Department of Medicine, Penn State University College of Medicine, Hershey, PA, USA

    Kristin Berger & Jeffrey J. Pu

  3. Department of Pathology, Pennsylvania State University College of Medicine, Hershey, PA, USA

    Jeffrey J. Pu

  4. Penn State Hershey Cancer Institute, Pennsylvania State University College of Medicine, Hershey, PA, USA

    Jeffrey J. Pu

Authors
  1. Lindsey Shantzer
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  2. Kristin Berger
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  3. Jeffrey J. Pu
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Correspondence to Jeffrey J. Pu.

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Shantzer, L., Berger, K. & Pu, J.J. Primary myelofibrosis and its targeted therapy. Ann Hematol 96, 531–535 (2017). https://doi.org/10.1007/s00277-016-2785-9

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  • DOI: https://doi.org/10.1007/s00277-016-2785-9

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