ASCO 2025: Rusfertide for Polycythemia Vera

Polycythemia vera (PV) is a rare blood cancer caused by a mutation in the JAK2 gene. It leads to the overproduction of red blood cells, raising the risk of blood clots and other cancers. 

Between 1999 and 2020, over 21,000 PV-related deaths were reported. The mortality rate declined until 2015 (from 0.4 to 0.22 per 100,000 people), then increased to 0.28 by 2020. Men, especially those over 85, non-Hispanic White individuals, and those living in the Midwest or rural areas had higher death rates. These findings show clear differences in PV-related death trends depending on demographics and geography, highlighting the importance of tailored treatment approaches.

Could a Self-Injected Therapy Help Reduce the Burden of Polycythemia Vera?

At the American Society of Clinical Oncology (ASCO) conference, research findings for polycythemia vera (PV) were presented. In this article, you’ll learn how rusfertide may help people with PV reduce the need for frequent blood draws (phlebotomies), better control blood levels, and improve common PV symptoms.

A New Way to Manage Blood Levels in PV

Rusfertide is a new type of therapy called a hepcidin mimetic. Hepcidin is a natural hormone that helps regulate iron levels in the body. Rusfertide blocks the movement of iron into the bone marrow, which reduces the body’s ability to make too many red blood cells.

The treatment is taken as a subcutaneous injection, meaning it’s administered under the skin and can be done at home. This may offer more independence for people who previously had to visit a clinic for regular procedures.

In people with PV, the body produces too many red blood cells, raising the risk of blood clots and heart problems. To manage this, many patients regularly undergo phlebotomy, a procedure where blood is removed to keep levels safe.

In phase 3 of the VERIFY trial, rusfertide was tested in PV patients who relied on frequent phlebotomies. Learn the results of this study below!

Key Trial Findings: Fewer Phlebotomies, Better Symptom Relief

The VERIFY trial enrolled 293 PV patients who depended on phlebotomy to control their high red blood cell counts. Some received rusfertide, while others received a placebo (inactive treatment), and both groups could continue other standard therapies.

Here are the results from the first 32 weeks:

• 76.9% of patients on rusfertide had no phlebotomy needs from weeks 20 to 32, compared to 32.9% with placebo.
• 72.8% of patients on rusfertide needed no phlebotomies at all during the entire 32 weeks, compared to 21.9% with placebo.
• 62.6% of patients using rusfertide had red blood cell counts (hematocrit) below 45% versus 14.4% on placebo.
• Patients also reported relief from symptoms such as night sweats, itching, abdominal discomfort, and bone pain.

Researchers found that patients who used rusfertide needed fewer phlebotomies and kept their blood levels better controlled. These results suggest that rusfertide could become an add-on treatment to existing therapies like hydroxyurea, interferon, or ruxolitinib. 

Side Effects and Safety Profile

As with any therapy, side effects were reported:

• Injection site reactions were more common with rusfertide (55.9%) compared to placebo (32.9%).
• Anemia occurred in 15.9% of patients on rusfertide, compared to 4.1% on placebo.
• Fatigue was reported at similar rates in both groups (about 15%).
• Importantly, serious side effects were rare and not considered related to the therapy.

Next Steps and Why This Matters for PV Patients

The VERIFY trial is continuing to study rusfertide in an open-label phase to see how long the benefits last. If approved, rusfertide could become part of the standard approach for managing PV, especially in patients who depend heavily on phlebotomies or experience major symptoms despite current treatments.

These findings are notably important given that PV-related deaths had been declining but began increasing again after 2015, affecting mostly older adults, non-Hispanic White individuals, and those living in rural areas. Having more treatment options tailored to patients’ real-life needs could help address these gaps. 

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Sources: 

• Trends and disparities in polycythemia vera–related mortality in the United States from 1999 to 2020
• VERIFY: Novel Polycythemia Vera Treatment Reduces Disease Burden, Improves Quality of Life in Patients With Phlebotomy-Dependent Disease