Emerging drugs for essential thrombocythemia

ABSTRACT

Introduction: Despite our recent progress in the understanding of essential thrombocythemia (ET) pathogenesis, the therapeutic management of this disease has remained largely unchanged in the past decades. Treatment has mostly focused on decreasing the risk of complications, especially prevention of thrombotic or hemorrhagic events.

Areas covered: Over recent years, the treatment options of ET have been expanding with some novel agents on the horizon. The classes of agents described in this review include targeted and immunomodulatory agents, such as JAK1/2 inhibitors, interferon-α, histone deacetylase inhibitors, telomerase inhibitors and human double minute 2 inhibitors. These compounds entered various stages of development, albeit the only portion of them is currently actively undergoing evaluation in clinical trials. In this review, we look at the current therapies and discuss novel agents available in the management of ET.

Expert opinion: The drug development in ET possesses several challenges stemming from its relatively benign and prolonged disease course. Therapy focused on reducing the risk of thrombotic and hemorrhagic complications and symptom management needs to be chosen wisely as a vast majority of these patients have a near-normal life expectancy. To date, no therapy has shown effective and definitive alteration of the disease behavior. Although novel agents are in development and hopefully some of them will extend treatment armamentarium of ET, their exact role remains to be determined.

KEYWORDS:

• Essential thrombocytopenia
• interferon
• JAK2 inhibitors
• myeloproliferative neoplasms
• novel therapies

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

A reviewer on this manuscript has disclosed that they are an investigator for PT1, MAJIC and EXEL trials. A second reviewer has disclosed that they are an advisor of AOP Pharmaceuticals Vienna, Austria. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Author Contributions

Both authors have reviewed the data, wrote and approved the final manuscript.

Additional information

Funding

This review was supported in part by  the National Cancer Institute, the MD Anderson Cancer Center Support Grant (CCSG) P30 CA016672.