Ep. 2: Integrating Rusfertide Into the Polycythemia Vera Treatment Algorithm
Key Points
- Rusfertide demonstrated applicability across patients with polycythemia vera (PV), regardless of background cytoreductive therapy.
- VERIFY data show durable hematocrit control, reduced phlebotomy burden, and improved quality-of-life measures.
- Injection site reactions were the most common adverse event and were generally mild, manageable, and decreased over time.
During a Clinical Insights discussion focused on polycythemia vera (PV), Oncology Brothers hosts Rahul Gosain, MD, MBA, of Wilmot Cancer Institute, and Rohit Gosain, MD, of Roswell Park Comprehensive Cancer Center, explored how emerging data may translate into real-world practice. Panelists Talha Badar, MBBS, MD, of Mayo Clinic; Aaron Gerds, MD, of Cleveland Clinic; and Andrew Kuykendall, MD, of Moffitt Cancer Center, discussed where the hepcidin mimetic rusfertide may fit within the current PV treatment paradigm if approved, with updates from the 2025 ASH Annual Meeting (ASH 2025) informing the conversation.
Clinical Positioning, Quality of Life, and Safety Considerations
Panelists noted that one of the strengths of the VERIFY trial design was its real-world applicability. Rusfertide was evaluated across a broad PV population, independent of background cytoreductive therapy, including patients receiving hydroxyurea, interferon, or no cytoreduction. This flexibility supports its potential use as an adjunct for patients with persistent hematocrit elevation, ongoing phlebotomy requirements, iron deficiency–related symptoms, or suboptimal response to standard therapies.
Updated data presented at ASH 2025 demonstrated that more than 70% of patients achieved durable hematocrit control, with meaningful improvements in patient-reported outcomes. The panel highlighted the relevance of these findings for older patients and those with limited social support, for whom frequent phlebotomy visits can be particularly burdensome. Maintaining hematocrit control while reducing clinic visits and symptom burden was a significant potential advantage.
From a safety perspective, rusfertide is administered as a self-injected subcutaneous therapy. Injection site reactions were the most common adverse event, occurring in both treatment and placebo arms, and were generally low grade and manageable with supportive measures such as topical corticosteroids and antihistamines.
Rates of injection site reactions decreased over time among patients continuing therapy. Mild anemia was observed, consistent with the drug’s mechanism of action, but was generally manageable. Although increases in platelet counts were noted, panelists emphasized that platelet count alone has not been clearly linked to thrombotic risk in PV, and no new safety signals emerged. High treatment retention across clinical studies further supported overall tolerability and patient acceptance.