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Essential Thrombocythemia in Adolescents and Young Adults: Clinical Aspects, Treatment Options and Unmet Medical Needs

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Opinion statement

Current treatment of essential thrombocythemia (ET) should primarily prevent thrombo-hemorrhagic events, without increasing the rate of fibrotic progression or leukemic evolution, and secondarily control microvascular symptoms. Unlike other classic BCR::ABL1-negative myeloproliferative neoplasms, ET is frequently diagnosed in adolescents and young adults (AYA), defined as individuals aged 15 to 39 years, in up to 20% of patients. However, since the current risk stratification of this disease is based on models, including that of ELN, IPSET-Thrombosis and its revised version, mainly applied to an older patients’ population, international guidelines are needed that specifically consider how to evaluate the prognosis of AYAs with ET. Furthermore, although ET is the most frequent MPN among AYA subjects, there is a lack of specific recommendations on how to treat it in this subgroup of patients, as management decisions are typically extrapolated from those for the elderly. Accordingly, since AYAs with ET represent a unique disease subset defined by attenuated genetic risk, more indolent phenotype, and longer survival than their older counterparts, treatment selection requires special attention to specific issues such as the risk of fibrotic/leukemic transformation, carcinogenicity, and fertility. This review article will provide a comprehensive overview of the diagnosis, prognostic stratification, and possible therapeutic approaches for AYA patients with ET, including antiplatelets/anticoagulants and cytoreductive agents, with a focus on pregnancy management in real-life clinical practice.

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Authors and Affiliations

  1. Hematology Division, Foundation IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Via Francesco Sforza 35, 20122, Milan, Italy

    Alessandra Iurlo MD, PhD, Cristina Bucelli MD & Daniele Cattaneo MD

  2. Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy

    Daniele Cattaneo MD

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  1. Alessandra Iurlo MD, PhD
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  2. Cristina Bucelli MD
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  3. Daniele Cattaneo MD
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Correspondence to Alessandra Iurlo MD, PhD.

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Iurlo, A., Bucelli, C. & Cattaneo, D. Essential Thrombocythemia in Adolescents and Young Adults: Clinical Aspects, Treatment Options and Unmet Medical Needs. Curr. Treat. Options in Oncol. 24, 802–820 (2023). https://doi.org/10.1007/s11864-023-01099-8

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