Novel Concepts of Treatment for Patients with Myelofibrosis and Related Neoplasms
Janus kinase (JAK) inhibition forms the cornerstone of the treatment of myelofibrosis (MF), and the JAK inhibitor ruxolitinib is often used as a second-line agent in patients with polycythemia vera (PV) who fail hydroxyurea (HU). In addition, ruxolitinib continues to be studied in patients with esse...
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-10-01
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| Series: | Cancers |
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| Online Access: | https://www.mdpi.com/2072-6694/12/10/2891 |
| _version_ | 1797551525479841792 |
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| author | Prithviraj Bose Lucia Masarova Srdan Verstovsek |
| author_facet | Prithviraj Bose Lucia Masarova Srdan Verstovsek |
| author_sort | Prithviraj Bose |
| collection | DOAJ |
| description | Janus kinase (JAK) inhibition forms the cornerstone of the treatment of myelofibrosis (MF), and the JAK inhibitor ruxolitinib is often used as a second-line agent in patients with polycythemia vera (PV) who fail hydroxyurea (HU). In addition, ruxolitinib continues to be studied in patients with essential thrombocythemia (ET). The benefits of JAK inhibition in terms of splenomegaly and symptoms in patients with MF are undeniable, and ruxolitinib prolongs the survival of persons with higher risk MF. Despite this, however, “disease-modifying” effects of JAK inhibitors in MF, i.e., bone marrow fibrosis and mutant allele burden reduction, are limited. Similarly, in HU-resistant/intolerant PV, while ruxolitinib provides excellent control of the hematocrit, symptoms and splenomegaly, reduction in the rate of thromboembolic events has not been convincingly demonstrated. Furthermore, JAK inhibitors do not prevent disease evolution to MF or acute myeloid leukemia (AML). Frontline cytoreductive therapy for PV generally comprises HU and interferons, which have their own limitations. Numerous novel agents, representing diverse mechanisms of action, are in development for the treatment of these three classic myeloproliferative neoplasms (MPNs). JAK inhibitor-based combinations, all of which are currently under study for MF, have been covered elsewhere in this issue. In this article, we focus on agents that have been studied as monotherapy in patients with MF, generally after JAK inhibitor resistance/intolerance, as well as several novel compounds in development for PV/ET. |
| first_indexed | 2024-03-10T15:47:20Z |
| format | Article |
| id | doaj.art-f65e4618ec154327b49c936991cb0ea4 |
| institution | Directory Open Access Journal |
| issn | 2072-6694 |
| language | English |
| last_indexed | 2024-03-10T15:47:20Z |
| publishDate | 2020-10-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Cancers |
| spelling | doaj.art-f65e4618ec154327b49c936991cb0ea42023-11-20T16:25:56ZengMDPI AGCancers2072-66942020-10-011210289110.3390/cancers12102891Novel Concepts of Treatment for Patients with Myelofibrosis and Related NeoplasmsPrithviraj Bose0Lucia Masarova1Srdan Verstovsek2Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USADepartment of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USAJanus kinase (JAK) inhibition forms the cornerstone of the treatment of myelofibrosis (MF), and the JAK inhibitor ruxolitinib is often used as a second-line agent in patients with polycythemia vera (PV) who fail hydroxyurea (HU). In addition, ruxolitinib continues to be studied in patients with essential thrombocythemia (ET). The benefits of JAK inhibition in terms of splenomegaly and symptoms in patients with MF are undeniable, and ruxolitinib prolongs the survival of persons with higher risk MF. Despite this, however, “disease-modifying” effects of JAK inhibitors in MF, i.e., bone marrow fibrosis and mutant allele burden reduction, are limited. Similarly, in HU-resistant/intolerant PV, while ruxolitinib provides excellent control of the hematocrit, symptoms and splenomegaly, reduction in the rate of thromboembolic events has not been convincingly demonstrated. Furthermore, JAK inhibitors do not prevent disease evolution to MF or acute myeloid leukemia (AML). Frontline cytoreductive therapy for PV generally comprises HU and interferons, which have their own limitations. Numerous novel agents, representing diverse mechanisms of action, are in development for the treatment of these three classic myeloproliferative neoplasms (MPNs). JAK inhibitor-based combinations, all of which are currently under study for MF, have been covered elsewhere in this issue. In this article, we focus on agents that have been studied as monotherapy in patients with MF, generally after JAK inhibitor resistance/intolerance, as well as several novel compounds in development for PV/ET.https://www.mdpi.com/2072-6694/12/10/2891imetelstatPRM-151KRT-232CPI-0610ropeginterferon alfa-2b |
| spellingShingle | Prithviraj Bose Lucia Masarova Srdan Verstovsek Novel Concepts of Treatment for Patients with Myelofibrosis and Related Neoplasms Cancers imetelstat PRM-151 KRT-232 CPI-0610 ropeginterferon alfa-2b |
| title | Novel Concepts of Treatment for Patients with Myelofibrosis and Related Neoplasms |
| title_full | Novel Concepts of Treatment for Patients with Myelofibrosis and Related Neoplasms |
| title_fullStr | Novel Concepts of Treatment for Patients with Myelofibrosis and Related Neoplasms |
| title_full_unstemmed | Novel Concepts of Treatment for Patients with Myelofibrosis and Related Neoplasms |
| title_short | Novel Concepts of Treatment for Patients with Myelofibrosis and Related Neoplasms |
| title_sort | novel concepts of treatment for patients with myelofibrosis and related neoplasms |
| topic | imetelstat PRM-151 KRT-232 CPI-0610 ropeginterferon alfa-2b |
| url | https://www.mdpi.com/2072-6694/12/10/2891 |
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