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Polycythemia vera: historical oversights, diagnostic details, and therapeutic views

Polycythemia vera (PV) is a relatively indolent myeloid neoplasm with median survival that exceeds 35 years in young patients, but its natural history might be interrupted by thrombotic, fibrotic, or leukemic events, with respective 20-year rates of 26%, 16%, and 4%. Current treatment strategies in...

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Autores principales: Tefferi, Ayalew, Vannucchi, Alessandro M., Barbui, Tiziano
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8632660/
https://www.ncbi.nlm.nih.gov/pubmed/34480106
http://dx.doi.org/10.1038/s41375-021-01401-3
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author Tefferi, Ayalew
Vannucchi, Alessandro M.
Barbui, Tiziano
author_facet Tefferi, Ayalew
Vannucchi, Alessandro M.
Barbui, Tiziano
author_sort Tefferi, Ayalew
collection PubMed
description Polycythemia vera (PV) is a relatively indolent myeloid neoplasm with median survival that exceeds 35 years in young patients, but its natural history might be interrupted by thrombotic, fibrotic, or leukemic events, with respective 20-year rates of 26%, 16%, and 4%. Current treatment strategies in PV have not been shown to prolong survival or lessen the risk of leukemic or fibrotic progression and instead are directed at preventing thrombotic complications. In the latter regard, two risk categories are considered: high (age >60 years or thrombosis history) and low (absence of both risk factors). All patients require phlebotomy to keep hematocrit below 45% and once-daily low-dose aspirin, in the absence of contraindications. Cytoreductive therapy is recommended for high-risk or symptomatic low-risk disease; our first-line drug of choice in this regard is hydroxyurea but we consider pegylated interferon as an alternative in certain situations, including in young women of reproductive age, in patients manifesting intolerance or resistance to hydroxyurea therapy, and in situations where treatment is indicated for curbing phlebotomy requirement rather than preventing thrombosis. Additional treatment options include busulfan and ruxolitinib; the former is preferred in older patients and the latter in the presence of symptoms reminiscent of post-PV myelofibrosis or protracted pruritus. Our drug choices reflect our appreciation for long-term track record of safety, evidence for reduction of thrombosis risk, and broader suppression of myeloproliferation. Controlled studies are needed to clarify the added value of twice- vs once-daily aspirin dosing and direct oral anticoagulants. In this invited review, we discuss our current approach to diagnosis, prognostication, and treatment of PV in general, as well as during specific situations, including pregnancy and splanchnic vein thrombosis.
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spelling pubmed-86326602021-12-10 Polycythemia vera: historical oversights, diagnostic details, and therapeutic views Tefferi, Ayalew Vannucchi, Alessandro M. Barbui, Tiziano Leukemia Review Article Polycythemia vera (PV) is a relatively indolent myeloid neoplasm with median survival that exceeds 35 years in young patients, but its natural history might be interrupted by thrombotic, fibrotic, or leukemic events, with respective 20-year rates of 26%, 16%, and 4%. Current treatment strategies in PV have not been shown to prolong survival or lessen the risk of leukemic or fibrotic progression and instead are directed at preventing thrombotic complications. In the latter regard, two risk categories are considered: high (age >60 years or thrombosis history) and low (absence of both risk factors). All patients require phlebotomy to keep hematocrit below 45% and once-daily low-dose aspirin, in the absence of contraindications. Cytoreductive therapy is recommended for high-risk or symptomatic low-risk disease; our first-line drug of choice in this regard is hydroxyurea but we consider pegylated interferon as an alternative in certain situations, including in young women of reproductive age, in patients manifesting intolerance or resistance to hydroxyurea therapy, and in situations where treatment is indicated for curbing phlebotomy requirement rather than preventing thrombosis. Additional treatment options include busulfan and ruxolitinib; the former is preferred in older patients and the latter in the presence of symptoms reminiscent of post-PV myelofibrosis or protracted pruritus. Our drug choices reflect our appreciation for long-term track record of safety, evidence for reduction of thrombosis risk, and broader suppression of myeloproliferation. Controlled studies are needed to clarify the added value of twice- vs once-daily aspirin dosing and direct oral anticoagulants. In this invited review, we discuss our current approach to diagnosis, prognostication, and treatment of PV in general, as well as during specific situations, including pregnancy and splanchnic vein thrombosis. Nature Publishing Group UK 2021-09-03 2021 /pmc/articles/PMC8632660/ /pubmed/34480106 http://dx.doi.org/10.1038/s41375-021-01401-3 Text en © The Author(s) 2021 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) .
spellingShingle Review Article
Tefferi, Ayalew
Vannucchi, Alessandro M.
Barbui, Tiziano
Polycythemia vera: historical oversights, diagnostic details, and therapeutic views
title Polycythemia vera: historical oversights, diagnostic details, and therapeutic views
title_full Polycythemia vera: historical oversights, diagnostic details, and therapeutic views
title_fullStr Polycythemia vera: historical oversights, diagnostic details, and therapeutic views
title_full_unstemmed Polycythemia vera: historical oversights, diagnostic details, and therapeutic views
title_short Polycythemia vera: historical oversights, diagnostic details, and therapeutic views
title_sort polycythemia vera: historical oversights, diagnostic details, and therapeutic views
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8632660/
https://www.ncbi.nlm.nih.gov/pubmed/34480106
http://dx.doi.org/10.1038/s41375-021-01401-3
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