Polycythemia vera is a classic myeloproliferative neoplasm and a chronic type of leukemia, which often leads to overproduction of various blood cells.
Summary: Managing Polycythemia Vera to Reduce Risks and Improve Lives
Polycythemia vera (PV) is a chronic myeloproliferative neoplasm-a type of blood cancer-characterized primarily by the overproduction of red blood cells, but often also involving elevated white blood cells and platelets. This cellular excess increases blood viscosity, leading to a heightened risk of both thrombotic (clotting) and bleeding events, such as heart attacks, strokes, and pulmonary embolisms. These complications can significantly reduce both life expectancy and quality of life for affected patients[1].
Key Risks and Symptoms
The main clinical risks in PV are blood clots (thrombosis) and bleeding, which can be life-threatening.
Patients also frequently experience inflammatory symptoms, including severe itching, fevers, night sweats, bone pain, and chronic fatigue, all of which further impair quality of life[1].
Treatment Goals and Approaches
The primary goals of PV management are to reduce the risk of thrombosis and to improve quality of life by controlling disease-related symptoms[1].
Standard treatments include:
Low-dose aspirin to reduce clotting risk.
Therapeutic phlebotomy, which involves regularly removing blood to lower hematocrit (the proportion of red blood cells in blood) and thus decrease blood thickness and clotting risk.
Maintaining hematocrit below 45% is a widely accepted target to minimize thrombotic events[1][2].
For patients at higher risk or with more severe disease, cytoreductive therapies are used to lower blood cell counts:
Ruxolitinib (Jakafi) and ropeginterferon alfa-2b-njft (Besremi) are FDA-approved medications for PV.
Rusfertide, a hepcidin mimetic, is currently in advanced clinical trials and has received special designations from the FDA due to its potential to reduce the need for phlebotomy and better control hematocrit[1][3][4].
Quality of Life Considerations
While controlling blood counts and reducing clot risk are central, managing chronic symptoms and minimizing the burden of frequent medical interventions (like phlebotomy) are increasingly recognized as important for patient well-being[5].
Newer therapies under investigation aim to reduce both the risk of complications and the chronic symptom burden, potentially allowing patients to maintain a near-normal life expectancy and improved daily functioning[5][1].
Conclusion
Effective management of polycythemia vera requires a dual focus: preventing life-threatening thrombotic events and optimizing quality of life by controlling symptoms and minimizing treatment burdens. Ongoing research and new therapeutic options hold promise for further improving outcomes for patients with this chronic condition[1][5].
References
Managing Polycythemia Vera to Reduce Risks and Improve Lives
Understanding Hematocrit Thresholds in Polycythemia Vera Treatment
Rusfertide Cuts Phlebotomy Need in Polycythemia Vera: Andrew Kuykendall, MD
Reducing Polycythemia Vera–Associated Thrombotic Risk Through Iron Regulation
Polycythemia Vera Management: Addressing the Burden of Symptoms and Phlebotomy Dependence
www.ajmc.com
Polycythemia vera is a classic myeloproliferative neoplasm and a chronic type of leukemia, which often leads to overproduction of various blood cells. Several medications are approved to treat this condition—among them ruxolitinib (Jakafi; Incyte), in December 2014,1 and ropeginterferon alfa-2b-njft (Besremi; PharmaEssentia), in November 20212—and others remain in clinical development. Rusfertide (Takeda) is currently being investigated in the phase 3 VERIFY trial (NCT05210790), with an estimate study complete date of June 2025.3 The hepcidin mimetic has already received breakthrough therapy, orphan drug, and fast track designations from the FDA.
In this interview, Andrew Kuykendall, MD, clinical researcher at Moffitt Cancer Center and a VERIFY investigator, breaks down how polycythemia vera manifests and common ways to reduce its negative impact on patient quality of life while reducing the risk of clinically worsening events.
Summary: Managing Polycythemia Vera to Reduce Risks and Improve Lives
Polycythemia vera (PV) is a chronic myeloproliferative neoplasm-a type of blood cancer-characterized primarily by the overproduction of red blood cells, but often also involving elevated white blood cells and platelets. This cellular excess increases blood viscosity, leading to a heightened risk of both thrombotic (clotting) and bleeding events, such as heart attacks, strokes, and pulmonary embolisms. These complications can significantly reduce both life expectancy and quality of life for affected patients[1].
Key Risks and Symptoms
The main clinical risks in PV are blood clots (thrombosis) and bleeding, which can be life-threatening.
Patients also frequently experience inflammatory symptoms, including severe itching, fevers, night sweats, bone pain, and chronic fatigue, all of which further impair quality of life[1].
Treatment Goals and Approaches
The primary goals of PV management are to reduce the risk of thrombosis and to improve quality of life by controlling disease-related symptoms[1].
Standard treatments include:
Low-dose aspirin to reduce clotting risk.
Therapeutic phlebotomy, which involves regularly removing blood to lower hematocrit (the proportion of red blood cells in blood) and thus decrease blood thickness and clotting risk.
Maintaining hematocrit below 45% is a widely accepted target to minimize thrombotic events[1][2].
For patients at higher risk or with more severe disease, cytoreductive therapies are used to lower blood cell counts:
Ruxolitinib (Jakafi) and ropeginterferon alfa-2b-njft (Besremi) are FDA-approved medications for PV.
Rusfertide, a hepcidin mimetic, is currently in advanced clinical trials and has received special designations from the FDA due to its potential to reduce the need for phlebotomy and better control hematocrit[1][3][4].
Quality of Life Considerations
While controlling blood counts and reducing clot risk are central, managing chronic symptoms and minimizing the burden of frequent medical interventions (like phlebotomy) are increasingly recognized as important for patient well-being[5].
Newer therapies under investigation aim to reduce both the risk of complications and the chronic symptom burden, potentially allowing patients to maintain a near-normal life expectancy and improved daily functioning[5][1].
Conclusion
Effective management of polycythemia vera requires a dual focus: preventing life-threatening thrombotic events and optimizing quality of life by controlling symptoms and minimizing treatment burdens. Ongoing research and new therapeutic options hold promise for further improving outcomes for patients with this chronic condition[1][5].
References
Managing Polycythemia Vera to Reduce Risks and Improve Lives
Understanding Hematocrit Thresholds in Polycythemia Vera Treatment
Rusfertide Cuts Phlebotomy Need in Polycythemia Vera: Andrew Kuykendall, MD
Reducing Polycythemia Vera–Associated Thrombotic Risk Through Iron Regulation
Polycythemia Vera Management: Addressing the Burden of Symptoms and Phlebotomy Dependence
Managing Polycythemia Vera to Reduce Risks and Improve Lives
Polycythemia vera is a classic myeloproliferative neoplasm and a chronic type of leukemia, which often leads to overproduction of various blood cells.
Polycythemia vera is a classic myeloproliferative neoplasm and a chronic type of leukemia, which often leads to overproduction of various blood cells. Several medications are approved to treat this condition—among them ruxolitinib (Jakafi; Incyte), in December 2014,1 and ropeginterferon alfa-2b-njft (Besremi; PharmaEssentia), in November 20212—and others remain in clinical development. Rusfertide (Takeda) is currently being investigated in the phase 3 VERIFY trial (NCT05210790), with an estimate study complete date of June 2025.3 The hepcidin mimetic has already received breakthrough therapy, orphan drug, and fast track designations from the FDA.
In this interview, Andrew Kuykendall, MD, clinical researcher at Moffitt Cancer Center and a VERIFY investigator, breaks down how polycythemia vera manifests and common ways to reduce its negative impact on patient quality of life while reducing the risk of clinically worsening events.
Last edited by a moderator:
