New Perspectives on Polycythemia Vera: From Diagnosis to Therapy
Polycythemia vera (PV) is mainly characterized by elevated blood cell counts, thrombotic as well as hemorrhagic predisposition, a variety of symptoms, and cumulative risks of fibrotic progression and/or leukemic evolution over time. Major changes to its diagnostic criteria were made in the 2016 revi...
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-08-01
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| Series: | International Journal of Molecular Sciences |
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| Online Access: | https://www.mdpi.com/1422-0067/21/16/5805 |
| _version_ | 1797558465400406016 |
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| author | Alessandra Iurlo Daniele Cattaneo Cristina Bucelli Luca Baldini |
| author_facet | Alessandra Iurlo Daniele Cattaneo Cristina Bucelli Luca Baldini |
| author_sort | Alessandra Iurlo |
| collection | DOAJ |
| description | Polycythemia vera (PV) is mainly characterized by elevated blood cell counts, thrombotic as well as hemorrhagic predisposition, a variety of symptoms, and cumulative risks of fibrotic progression and/or leukemic evolution over time. Major changes to its diagnostic criteria were made in the 2016 revision of the World Health Organization (WHO) classification, with both hemoglobin and hematocrit diagnostic thresholds lowered to 16.5 g/dL and 49% for men, and 16 g/dL and 48% for women, respectively. The main reason leading to these changes was represented by the recognition of a new entity, namely the so-called “masked PV”, as individuals suffering from this condition have a worse outcome, possibly owing to missed or delayed diagnoses and lower intensity of treatment. Thrombotic risk stratification is of crucial importance to evaluate patients’ prognosis at diagnosis. Currently, patients are stratified into a low-risk group, in the case of younger age (<60 years) and no previous thromboses, and a high-risk group, in the case of patients older than 60 years and/or with a previous thrombotic complication. Furthermore, even though they have not yet been formally included in a scoring system, generic cardiovascular risk factors, particularly hypertension, smoking, and leukocytosis, contribute to the thrombotic overall risk. In the absence of agents proven to modify its natural history and prevent progression, PV management has primarily been focused on minimizing the thrombotic risk, representing the main cause of morbidity and mortality. When cytoreduction is necessary, conventional therapies include hydroxyurea as a first-line treatment and ruxolitinib and interferon in resistant/intolerant cases. Each therapy, however, is burdened by specific drawbacks, underlying the need for improved strategies. Currently, the therapeutic landscape for PV is still expanding, and includes several molecules that are under investigation, like long-acting pegylated interferon alpha-2b, histone deacetylase inhibitors, and murine double minute 2 (MDM2) inhibitors. |
| first_indexed | 2024-03-10T17:31:46Z |
| format | Article |
| id | doaj.art-9757570a405345f791099620b5e0f716 |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-10T17:31:46Z |
| publishDate | 2020-08-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-9757570a405345f791099620b5e0f7162023-11-20T10:00:02ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672020-08-012116580510.3390/ijms21165805New Perspectives on Polycythemia Vera: From Diagnosis to TherapyAlessandra Iurlo0Daniele Cattaneo1Cristina Bucelli2Luca Baldini3Hematology Division, Foundation IRCCS Ca’ Granda Ospedale Maggiore Policlinico, 20122 Milan, ItalyHematology Division, Foundation IRCCS Ca’ Granda Ospedale Maggiore Policlinico, 20122 Milan, ItalyHematology Division, Foundation IRCCS Ca’ Granda Ospedale Maggiore Policlinico, 20122 Milan, ItalyHematology Division, Foundation IRCCS Ca’ Granda Ospedale Maggiore Policlinico, 20122 Milan, ItalyPolycythemia vera (PV) is mainly characterized by elevated blood cell counts, thrombotic as well as hemorrhagic predisposition, a variety of symptoms, and cumulative risks of fibrotic progression and/or leukemic evolution over time. Major changes to its diagnostic criteria were made in the 2016 revision of the World Health Organization (WHO) classification, with both hemoglobin and hematocrit diagnostic thresholds lowered to 16.5 g/dL and 49% for men, and 16 g/dL and 48% for women, respectively. The main reason leading to these changes was represented by the recognition of a new entity, namely the so-called “masked PV”, as individuals suffering from this condition have a worse outcome, possibly owing to missed or delayed diagnoses and lower intensity of treatment. Thrombotic risk stratification is of crucial importance to evaluate patients’ prognosis at diagnosis. Currently, patients are stratified into a low-risk group, in the case of younger age (<60 years) and no previous thromboses, and a high-risk group, in the case of patients older than 60 years and/or with a previous thrombotic complication. Furthermore, even though they have not yet been formally included in a scoring system, generic cardiovascular risk factors, particularly hypertension, smoking, and leukocytosis, contribute to the thrombotic overall risk. In the absence of agents proven to modify its natural history and prevent progression, PV management has primarily been focused on minimizing the thrombotic risk, representing the main cause of morbidity and mortality. When cytoreduction is necessary, conventional therapies include hydroxyurea as a first-line treatment and ruxolitinib and interferon in resistant/intolerant cases. Each therapy, however, is burdened by specific drawbacks, underlying the need for improved strategies. Currently, the therapeutic landscape for PV is still expanding, and includes several molecules that are under investigation, like long-acting pegylated interferon alpha-2b, histone deacetylase inhibitors, and murine double minute 2 (MDM2) inhibitors.https://www.mdpi.com/1422-0067/21/16/5805polycythemia verarisk factorstarget therapyhydroxyurearuxolitinibinterferon |
| spellingShingle | Alessandra Iurlo Daniele Cattaneo Cristina Bucelli Luca Baldini New Perspectives on Polycythemia Vera: From Diagnosis to Therapy International Journal of Molecular Sciences polycythemia vera risk factors target therapy hydroxyurea ruxolitinib interferon |
| title | New Perspectives on Polycythemia Vera: From Diagnosis to Therapy |
| title_full | New Perspectives on Polycythemia Vera: From Diagnosis to Therapy |
| title_fullStr | New Perspectives on Polycythemia Vera: From Diagnosis to Therapy |
| title_full_unstemmed | New Perspectives on Polycythemia Vera: From Diagnosis to Therapy |
| title_short | New Perspectives on Polycythemia Vera: From Diagnosis to Therapy |
| title_sort | new perspectives on polycythemia vera from diagnosis to therapy |
| topic | polycythemia vera risk factors target therapy hydroxyurea ruxolitinib interferon |
| url | https://www.mdpi.com/1422-0067/21/16/5805 |
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