Uncontrolled PV is highlighted by a key opinion leader.
Aaron Gerds, MD, MS: Patients who are older or have a history of thrombosis, really the mainstay of treatment in addition to a low-dose aspirin or anticoagulation [drug] is really a medication to control the counts or what we…term [as] cytoreduction. That includes hydroxyurea and interferons in the frontline setting as well as ruxolitinib in the second-line study. That forms the crux of treatment for those high-risk patients.
The guidelines focus on the use of hydroxyurea interferons and ruxolitinib as the mainstay of therapy for polycythemia vera [PV] along with cytoreduction and, of course, antiplatelet agents like aspirin and or anticoagulation. But these aren’t perfect medications, and the disease can be uncontrolled otherwise. [With] uncontrolled polycythemia vera, you’ve gone through these agents and you’re still not getting the counts under control, you’re running into [adverse] effects and things are not optimally controlled. Now we want to think outside the guidelines and keep continuing to push. And that’s why we make an emphasis in the guidelines to recommend referrals to centers that have clinical trials. There are a number of ongoing clinical trials, not only in North America, but globally, for new drugs for polycythemia vera—new treatment approaches, new agents, very innovative [ones]…as well as other agents…that could potentially alter the face of the disease.… Across the board, there are a lot of very exciting trials ongoing in polycythemia vera. For patients who have uncontrolled disease, referring [them] to a place that has clinical trials is so important. That’s why we would try to emphasize that as an option in the guidelines. There are other agents that are…available to treat polycythemia vera. Outside the guideline triplet, if you will, of hydroxyurea, interferons, and ruxolitinib, we often think about busulfan, [which] does an incredibly great job of controlling counts at low doses in polycythemia vera. [It] is a pretty well-tolerated medication with a well-established [adverse] effect profile. The challenge, though, with this [is that] it’s an alkylating agent, and it can increase the risk of disease progression. For short periods of time it could be an option. But, again, because of the associated risk of disease progression to acute leukemia, a very aggressive form of acute leukemia, we’ve actually ended up removing that as a preferred option within the guidelines but know [it] can still be used. As a panel, we want to emphasize in those cases where the classic 3 medications don’t control the counts. We want to push for clinical trials, though, because that’s the only way to move the field forward.
Transcript is AI-generated and edited for clarity and readability.
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