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ASCO 2025 | A review of the clinical safety and efficacy of ruxolitinib 10 years after its approval for PV
Naveen Pemmaraju, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, comments on a recent review of the clinical safety and efficacy of ruxolitinib in polycythemia vera (PV), 10 years after its approval for this indication. Dr Pemmaraju highlights that ruxolitinib has shown encouraging efficacy in patients with PV, with no new safety signals observed. The improved event-free survival (EFS) demonstrated by the agent suggests that it may have a disease-modifying effect, an exciting prospect for clinicians managing the disease. This interview took place during the 2025 American Society of Clinical Oncology (ASCO) Meeting in Chicago, IL.
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Transcript
Another exciting area of the rare blood cancers is that of MPN or myeloproliferative neoplasms. The MPNs are further subdivided classically into three MPNs. So one is ET, essential thrombocytosis, another is polycythemia vera, PV, and then myelofibrosis.
In polycythemia vera, it’s one of these disorders that is now considered a blood cancer and has been around and has been known for hundreds of years with little to no development until very recently...
Another exciting area of the rare blood cancers is that of MPN or myeloproliferative neoplasms. The MPNs are further subdivided classically into three MPNs. So one is ET, essential thrombocytosis, another is polycythemia vera, PV, and then myelofibrosis.
In polycythemia vera, it’s one of these disorders that is now considered a blood cancer and has been around and has been known for hundreds of years with little to no development until very recently. So I was proud to put together this review article with my colleagues, which looks at a novel agent, a JAK inhibitor that targets P Vera. And that JAK inhibitor is known as ruxolitinib. It’s now been about 10 or 11 years since the initial approval. And so we wanted to review sort of the safety and indications.
Overall, we’re finding that the ruxolitinib agent as a JAK1, JAK2 inhibitor is highly effective in patients with polycythemia vera. And it has a US FDA approval of after hydroxyurea intolerance or resistance. So we reviewed those indications.
In terms of safety, there are some well-known safety signals, which we have documented before, which may include weight gain in some patients, shingles, opportunistic infections, and other infections in some patients, and even non-melanoma skin cancers such as basal cell and squamous cell. So those have been known in both myelofibrosis and P Vera. So no new safety signals in that regard.
But now 10 plus years after the approval, we’re starting to see studies come out, such as Claire Harrison’s MAJIC study, which was published in JCO in 2024, which really for the first time show that the ruxolitinib agent is improving EFS, which is event-free survival, a common marker used in solid tumors, but now making its way into blood cancers. And again, when you show improvement in EFS, it brings up the concept in question of improved disease modification. And that’s something we have not really talked about much in P Vera. And I’m really excited to see that. So with the advent of new agents, interferons, the JAK inhibitors, and other agents coming to P Vera, can we start talking about modifying the disease at the molecular level.
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