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Pharmacologic management of ET: established therapies and emerging agents

Rejowana Rouf1, Douglas Tremblay1

  • 1Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

Expert Opinion on Pharmacotherapy
|October 27, 2025
PubMed
Abstract

Insights

Established and emerging treatments for essential thrombocythemia (ET) are reviewed. Novel agents targeting specific mutations and pathways show promise for improving platelet counts and managing ET.

Area of Science:

  • Hematology
  • Oncology
  • Pharmacotherapy

Background:

  • Essential thrombocythemia (ET) is a myeloproliferative neoplasm characterized by elevated platelet counts.
  • Current cytoreductive therapies include hydroxyurea, interferon-α, and anagrelide.
  • Emerging data and clinical trials are expanding treatment options.

Purpose of the Study:

  • To review established cytoreductive strategies for ET.
  • To evaluate emerging targeted agents and novel therapeutic approaches.
  • To discuss future directions in ET management.

Main Methods:

  • Review of recent clinical trials (2018-2025) in ET.
  • Analysis of expert consensus and data from hematology meetings.
  • Evaluation of established agents (hydroxyurea, interferon-α, anagrelide) and novel targeted therapies.

Main Results:

  • Bomedemstat (LSD1 inhibitor) demonstrated significant platelet reduction and mutation burden improvement.
  • Ropeginterferon alfa-2b showed efficacy compared to anagrelide in a Phase 3 trial.
  • Emerging agents target JAK-STAT pathways and CALR mutations, showing potential for selective suppression of malignant hematopoiesis.

Conclusions:

  • Future ET management will integrate molecular risk stratification and combination regimens.
  • Personalized approaches using allele burden and symptom control will define next-generation ET pharmacotherapy.
  • Novel agents offer improved efficacy and targeted mechanisms for ET treatment.
Keywords:
AnagrelideETLSD1 inhibitorbomedemstatcytoreductive therapyhydroxyureainterferon-αropeginterferon alfa-2b

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