Rusfertide for Polycythemia Vera: What to Know
- Rusfertide is a new FDA‑approved treatment that helps people with polycythemia vera keep their hematocrit (red blood cell levels) below 45% and reduce how often they need phlebotomies, or blood removal.
- It works by limiting the iron the body uses to make red blood cells, giving patients a gentler alternative to frequent blood removal.
- In clinical trials, rusfertide helped many more patients maintain safe hematocrit levels and cut down on phlebotomies. People also reported feeling less fatigued and noticed improvements in other PV‑related symptoms.
- Rusfertide is generally well tolerated, with injection‑site irritation and occasional anemia being the most common side effects.
- “For patients, this may improve control of hematocrit, reduce the need for phlebotomies, and improve quality of life,” Dr. Michael Keng, a UVA Health hematologist, tells SurvivorNet.
“The recent FDA approval of rusfertide adds an important treatment option, particularly for patients who continue to require frequent phlebotomies [blood removal], which can worsen iron deficiency and its associated symptoms,” Dr. Jan Philipp Bewersdorf, an oncologist at Yale University, tells SurvivorNet.
Read MoreHow Rusfertide Works
Rusfertide works differently from most existing PV treatments. It acts like the natural hormone hepcidin, which regulates iron availability in the body. By reducing the amount of iron available, rusfertide slows the production of new red blood cells.It is given as a weekly injection under the skin.
Dr. Angela Fleischman, an oncologist at UC Irvine specializing in hematologic malignancies, notes that the updated NCCN guidelines give patients a much-needed new treatment approach.
“This update gives patients with polycythemia vera an alternative to repeated phlebotomy for controlling excess red blood cell production. It can help patients avoid the iron deficiency and associated symptoms caused by frequent phlebotomy, while also providing more consistent control of hematocrit over time,” Dr. Fleischman says.
In addition to improving control of hematocrit and reducing the need for phlebotomies, Dr. Michael Keng, a UVA Health hematologist, tells SurvivorNet the approach can “improve quality of life.”
The Data Behind the Approval
The approval was based on two clinical trials, REVIVE and VERIFY.
In VERIFY, 293 people with PV who needed frequent phlebotomies received rusfertide or placebo.
- About 77% of those receiving rusfertide achieved the study’s treatment goal, compared with 33% receiving placebo.
- About 63% were able to keep their hematocrit below 45%, compared with 14% receiving placebo.
- Rusfertide reduced the need for phlebotomies and improved fatigue and other PV-related symptoms.
“Treatment of PV aims to reduce the risk of thrombotic complications by maintaining a hematocrit below 45% through phlebotomy and, in selected patients, medications that reduce blood cell production,” Dr. Bewersdorf explains.
“In clinical trials, [rusfertide] improved hematocrit control, substantially reduced the need for phlebotomy, and improved fatigue and other disease-related symptoms.”
New NCCN Recommendations
The National Comprehensive Cancer Network (NCCN) now includes rusfertide as an option for both low- and high-risk PV.
For lower-risk patients, it may be used instead of or in addition to standard therapy when frequent phlebotomy is needed or poorly tolerated.
For higher-risk patients, it can be added to other medications when the hematocrit remains difficult to control, or phlebotomies are still frequently required.
More Expert Resources on Polycythemia Vera
Rusfertide does not necessarily replace other PV treatments, particularly when cytoreductive therapy is needed because of higher-risk disease, very high platelet or white blood cell counts, an enlarged spleen, or other disease-related problems.
Rusfertide is currently best viewed as a treatment that controls the effects of PV, particularly excess red blood cell production and the need for phlebotomy, rather than as a treatment that changes the underlying disease. It has not yet been shown to eliminate the abnormal blood-cell clone or prevent progression to myelofibrosis or acute leukemia.
What This Means for Patients
Researchers are continuing to study whether rusfertide affects the underlying disease or reduces long-term complications.
“The NCCN’s Category 1 recommendation for rusfertide is an exciting clinical advancement for people living with polycythemia vera. This is especially true for patients who require frequent phlebotomies in the setting of current therapies available,” Dr. Keng tells SurvivorNet.
Rusfertide has generally been well tolerated. The most common side effect was injection-site irritation, occurring in about 56% of patients in VERIFY. Anemia occurred in about 16%. Because rusfertide changes how the body uses and stores iron, doctors will monitor blood counts and iron studies during treatment.
For people living with PV, rusfertide is an additional option, particularly for those who continue to need frequent phlebotomies or have difficulty maintaining their hematocrit.
“These findings support rusfertide as an option for patients seeking better hematocrit control and a reduced phlebotomy burden,” Dr. Bewersdorf says. As rusfertide is used more widely, longer-term follow-up will help determine its effects on the overall course of PV.
Questions To Ask Your Doctor
- Is treatment with rusfertide recommended in my case?
- Will this approach reduce how often I need phlebotomies?
- How will I be monitored while taking rusfertide?
- What side effects should I be aware of?
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