TY - JOUR
T1 - SOHO State of the Art Update and Next Questions
T2 - Novel Therapies for Polycythemia Vera
AU - Masarova, Lucia
AU - Chifotides, Helen T.
N1 - Publisher Copyright:
© 2023 Elsevier Inc.
PY - 2024/3
Y1 - 2024/3
N2 - In the recent years, landmark advancements in the treatment of polycythemia vera (PV) have been achieved. We witnessed the regulatory approval of ropeginterferon and the advanced clinical development of other novel agents that may affect the underlying pathophysiological mechanisms of the disease. Agents with the potential of disease modification may soon overtake preceding treatment options that were based on the patient's age and history of thrombosis. Recent studies using ropeginterferon in low-risk PV patients earlier in the disease course challenge the current treatment paradigm and shift the focus on modifying the course of the disease. Hepcidin mimetics offer an excellent alternative to phlebotomy, providing better quality of life, and may lead to improved outcomes in PV by tight hematocrit control. Novel agents, such as histone deacetylase inhibitors, hold promise to complement the therapeutic landscape of PV and might be particularly promising in rationale combinations. Ruxolitinib is well established as an approved second-line treatment for PV. In the frontline setting, the precise role of ruxolitinib, which also represents an appealing agent in combination regimens, will be determined in ongoing research studies. Longer follow-up is necessary to assess whether novel agents/regimens elicit fewer thromboembolic/ hemorrhagic events and halt disease progression to myelofibrosis and acute myeloid leukemia. We aspire that disease-modifying approaches in PV are on the horizon, and that we will be empowered to ultimately change the natural course of the disease and profoundly impact the lives of PV patients in the near future.
AB - In the recent years, landmark advancements in the treatment of polycythemia vera (PV) have been achieved. We witnessed the regulatory approval of ropeginterferon and the advanced clinical development of other novel agents that may affect the underlying pathophysiological mechanisms of the disease. Agents with the potential of disease modification may soon overtake preceding treatment options that were based on the patient's age and history of thrombosis. Recent studies using ropeginterferon in low-risk PV patients earlier in the disease course challenge the current treatment paradigm and shift the focus on modifying the course of the disease. Hepcidin mimetics offer an excellent alternative to phlebotomy, providing better quality of life, and may lead to improved outcomes in PV by tight hematocrit control. Novel agents, such as histone deacetylase inhibitors, hold promise to complement the therapeutic landscape of PV and might be particularly promising in rationale combinations. Ruxolitinib is well established as an approved second-line treatment for PV. In the frontline setting, the precise role of ruxolitinib, which also represents an appealing agent in combination regimens, will be determined in ongoing research studies. Longer follow-up is necessary to assess whether novel agents/regimens elicit fewer thromboembolic/ hemorrhagic events and halt disease progression to myelofibrosis and acute myeloid leukemia. We aspire that disease-modifying approaches in PV are on the horizon, and that we will be empowered to ultimately change the natural course of the disease and profoundly impact the lives of PV patients in the near future.
KW - Disease modification
KW - Hepcidin mimetics
KW - Interferon
KW - Polycythemia vera
UR - http://www.scopus.com/inward/record.url?scp=85180606953&partnerID=8YFLogxK
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U2 - 10.1016/j.clml.2023.11.004
DO - 10.1016/j.clml.2023.11.004
M3 - Review article
C2 - 38135633
AN - SCOPUS:85180606953
SN - 2152-2650
VL - 24
SP - 141
EP - 148
JO - Clinical Lymphoma, Myeloma and Leukemia
JF - Clinical Lymphoma, Myeloma and Leukemia
IS - 3
ER -