Evolving Management Strategies for Phlebotomy-Dependent Polycythemia Vera: The Path Forward
Panelists discuss how new treatments like rusfertide (a hepcidin mimetic) offer promising options for polycythemia vera patients, demonstrating benefits in reducing phlebotomy requirements while addressing the paradoxical iron deficiency caused by current treatments, potentially improving quality of life while allowing patients to maintain their existing cytoreductive therapies, though questions remain about whether it will show the disease-modifying effects seen with ruxolitinib and interferons.
Summary of Rusfertide for Polycythemia Vera: VERIFY Clinical Trial
Novel Therapeutic Approach
- Mechanism of action: Rusfertide is a hepcidin mimetic that regulates iron metabolism
- Acts as a “chemical phlebotomy” by reducing erythrocytosis
- Contrasts with JAK inhibition approaches used in other myeloproliferative neoplasms
- VERIFY trial design (Phase 3):
- Patient population: Patients with polycythemia vera ≥18 years requiring phlebotomies due to inadequate hematocrit control
- Study design: Randomized placebo-controlled with open-label extension phases
- Treatment approach: Add-on to existing therapy (cytoreductive agents and/or phlebotomies)
- Primary and key secondary end points were met (details pending full publication)
Clinical Experience and Potential Benefits
- Phlebotomy reduction:
- Patients experienced rapid and noticeable decrease in phlebotomy requirements
- Self-evident efficacy improved adherence and satisfaction
- Iron homeostasis restoration:
- Addresses the unrecognized problem of iron deficiency in patients with polycythemia vera
- Iron deficiency from repeated phlebotomies causes significant symptomatology
- Improved iron stores may enhance quality of life while maintaining disease control
- Implementation considerations:
- Patient acceptance of self-injection appears manageable, particularly in centers familiar with interferon therapy
- Add-on approach allows continuation of existing therapies, enhancing patient comfort
- Quality of life benefits reported as key secondary end point
Future Directions
- Outstanding questions:
- Disease-modifying potential (impact on JAK2 allele burden) remains unknown
- Positioning in treatment algorithm yet to be determined
- Long-term safety and efficacy data forthcoming
The panel viewed rusfertide as an important addition to the PV treatment landscape, offering a novel mechanism to achieve both disease control and symptom improvement by addressing the iron deficiency that often accompanies traditional management approaches.
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