Emerging therapies for the treatment of essential thrombocythemia

Adolfo Enrique Diaz, Robin M. Scherber, Ruben Mesa

Research output: Contribution to journalReview article


Introduction: Historically the treatment of essential thrombocythemia (ET) has focused on decreasing the risk of thrombotic and hemorrhagic complications. Despite numerous available therapies with a long history of clinical use, there remains only one Federal Drug Administration approved therapy for high-risk ET patient populations. Areas covered: In recent years, the treatment landscape for ET has been evolving at a fast pace. Therapies such as JAK1/JAK2 inhibitors have demonstrated promising safety, tolerability, and efficacy. Alternative targeted agents are also in development, including Histone Deacetylase Inhibitors, Telomerase Inhibitors, and Human Double Minute 2 Inhibitors. Herein, authors provide an overview of emerging therapies for the treatment of ET. Expert opinion: Patients with ET often have a near-normal life expectancy. Treatments are targeted to primary and secondary prevention of complications such as thrombosis and hemorrhagic events. To date, no therapies have demonstrated a definitive alteration of the natural history of ET. Since several exciting agents are in development, well designed prospective trials would offer more facts regarding choice of therapy, sequence of use and/or association with established therapies.

Original languageEnglish (US)
Pages (from-to)567-575
Number of pages9
JournalExpert Opinion on Orphan Drugs
Issue number10
Publication statusPublished - Oct 3 2018
Externally publishedYes



  • essential thrombocythemia
  • interferon
  • JAK2 inhibitors
  • Myeloproliferative neoplasms
  • newer therapies

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)

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