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. 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.

This transcript has been lightly edited for clarity; captions were auto-generated.

Transcript

What are the major risks associated with polycythemia vera, and how do they impact patients’ quality of life?

Polycythemia vera is really one of these myeloproliferative neoplasms that leads to overproduction of a lot of different blood cells. Patients often present with too many blood cells: white blood cells, red blood cells, [or] platelets. It's really hallmarked by the overproduction of red blood cells. We know that patients with polycythemia vera are at a heightened risk for thrombotic and bleeding events. This is really something that can certainly shorten or reduce the quality of their lives if they endure one of these thrombotic events—be it an arterial event, such as a heart attack, a stroke, a blood clot, [or] a venous event, [which] could be like a pulmonary embolism or something like that—and in addition to this, there's also significant inflammatory symptoms that patients have as well with polycythemia vera, whether it's itching, fevers, chills, night sweats, bone pain, fatigue is another main one.

Really, our focus on treatment of polycythemia vera is to reduce the risk for thrombosis and to optimize quality of life through control of some of these disease-related symptoms. We achieve this in a variety of ways, but one of the main things that we do is we put patients on a baby aspirin and we try to keep their hematocrit—which is the percentage of blood made up by red blood cells—we try to keep that well controlled to kind of thin the blood, if you will. Typically, we've done this historically using therapeutic phlebotomies, where we basically bring patients in repeatedly and take blood from them until their blood counts go down low enough that we feel like they're at less of a risk for some of these thrombotic events.

References

1. FDA approves Jakafi (ruxolitinib) for the treatment of patients with uncontrolled polycythemia vera. News release. Incyte Corporation. December 4, 2014. Accessed March 5, 2025. https://investor.incyte.com/news-releases/news-release-details/fda-approves-jakafir-ruxolitinib-treatment-patients-uncontrolled#:~:text=WILMINGTON%2C%20Del.,and%20progressive%20blood%20cancer1

2. Rosa K. FDA approves ropeginterferon alfa-2b-njft for polycythemia vera. OncLive®. November 12, 2021. Accessed March 5, 2025. https://www.onclive.com/view/fda-approves-ropeginterferon-alfa-2b-njft-for-polycythemia-vera

3. A phase 3 study of rusfertide in patients with polycythemia vera. Clinicaltrials.gov. Updated July 17, 2017. Accessed March 5, 2025. https://clinicaltrials.gov/study/NCT05210790