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Ongoing Clinical Trials in Myeloproliferative Neoplasms

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Myeloproliferative Neoplasms

Part of the book series: Hematologic Malignancies ((HEMATOLOGIC))

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Abstract

There are many new drugs in clinical development for patients with classic Philadelphia-negative myeloproliferative neoplasms (Ph-negative MPNs). JAK2 inhibitors, developed to target the JAK2 enzyme which is found mutated in the majority of patients with Ph-negative MPNs, lead to reduction in splenomegaly, improvement in systemic symptoms and quality of life in patients with myelofibrosis (MF), while in patients with polycythemia vera (PV) and essential thrombocythemia (ET) who are intolerant or resistant to hydroxyurea, they may control blood cell count(s) and signs and symptoms of the disease. Currently, there are three phase III trials underway to better determine the activity of JAK2 inhibitor ruxolitinib in patients with MF and PV, and a number of phase I/II studies with other JAK2 inhibitors for patients with MF. Pomalidomide is a derivative of thalidomide with activity in improving the anemia in patients with MF and is being studied in a phase III placebo-controlled study for possible approval. Other compounds in clinical studies are histone deacetylase inhibitors, mTOR inhibitor, TGF-β inhibitor, and bisphosphonates. It can be safely said that the field of Ph-negative MPNs have gone from neglect to prominence, with new drugs in late stage development at the moment. In this chapter, we review some of the ongoing clinical trials for patients with Ph-negative MPNs.

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Authors and Affiliations

  1. Hematology and Oncology Department, Hospital Israelita Albert Einstein, São Paulo, SP, Brazil

    Fabio P. S. Santos

  2. Department of Leukemia, The University of Texas M.D. Anderson Cancer Center, Houston, TX, USA

    Srdan Verstovsek

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Correspondence to Srdan Verstovsek .

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Editors and Affiliations

  1. Div. Ematologia, Ospedali Riuniti di Bergamo, Largo Barozzi 1, Bergamo, 24128, Italy

    Tiziano Barbui

  2. Dept. Internal Medicine, Div. Hematology, Mayo Clinic, First Street SW. 200, Rochester, 55905, Minnesota, USA

    Ayalew Tefferi

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Santos, F.P.S., Verstovsek, S. (2012). Ongoing Clinical Trials in Myeloproliferative Neoplasms. In: Barbui, T., Tefferi, A. (eds) Myeloproliferative Neoplasms. Hematologic Malignancies. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-24989-1_18

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  • DOI: https://doi.org/10.1007/978-3-642-24989-1_18

  • Published:

  • Publisher Name: Springer, Berlin, Heidelberg

  • Print ISBN: 978-3-642-24988-4

  • Online ISBN: 978-3-642-24989-1

  • eBook Packages: MedicineMedicine (R0)

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