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Advances in Risk Stratification and Treatment of Polycythemia Vera and Essential Thrombocythemia

  • Myeloproliferative Neoplasms (P Bose, Section Editor)
  • Published:
Current Hematologic Malignancy Reports Aims and scope Submit manuscript

Abstract

Purpose of Review

Estimating and modifying thrombotic risk is currently the mainstay of care for patients with polycythemia vera (PV) and essential thrombocythemia (ET). In recent years, however, increased attention has shifted towards quality of life and disease modification. In this review, we discuss recent advances in risk stratification, present updated results for ruxolitinib and interferon randomized clinical trials, discuss new approaches in antiplatelet and anticoagulant treatment, and summarize early phase trials of novel agents and emerging therapeutic concepts for the treatment of PV and ET.

Recent Findings

International collaborations and novel technologies, i.e., next-generation sequencing and machine learning techniques, have demonstrated excellent abilities to improve thrombotic risk stratification in PV and ET. Updated results from ruxolitinib and interferon randomized clinical trials have confirmed excellent efficacy and safety of these agents, both as first- and second-line treatments. Early trials of novel agents (histone deacetylase inhibitors, telomerase inhibitors, lysine-specific demethylase-1 inhibitors, human double-minute 2 inhibitors, and hepcidin mimetics) have shown encouraging efficacy and safety in blood count control, reduction of splenomegaly, and alleviation of disease-related symptoms. Finally, accumulating evidence suggested that direct oral anticoagulants may be a valid therapeutic alternative to warfarin for prolonged thromboprophylaxis.

Summary

International collaborations (“big data”) with the help of new technologies represent an exciting new approach to analyze rare outcomes in rare diseases, especially for identifying novel prognostic biomarkers in PV and ET. Randomized clinical trials are also needed to fully elucidate whether novel agents may establish new standards of care.

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Authors and Affiliations

  1. Department of Internal Medicine, General Hospital of Sibenik-Knin County, Stjepana Radića 83, 22000, Sibenik, Croatia

    Ivan Krecak

  2. School of Medicine, University of Rijeka, Rijeka, Croatia

    Ivan Krecak

  3. Division of Hematology, University Hospital Dubrava, Zagreb, Croatia

    Marko Lucijanic

  4. School of Medicine, University of Zagreb, Zagreb, Croatia

    Marko Lucijanic

  5. Department of Leukemia, MD Anderson Cancer Center, Houston, TX, USA

    Srdan Verstovsek

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Krecak, I., Lucijanic, M. & Verstovsek, S. Advances in Risk Stratification and Treatment of Polycythemia Vera and Essential Thrombocythemia. Curr Hematol Malig Rep 17, 155–169 (2022). https://doi.org/10.1007/s11899-022-00670-8

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