Future Direction for Polycythemia Vera



Ruben Mesa, MD: Unmet needs in polycythemia vera include us having an understanding of why patients progress and being able to develop targeted approaches for leading to that inhibition of progression. I do believe that ruxolitinib, when effective, likely helps avoid progression. I believe that interferons, when effective, may have an impact on that as well. But one of our limiters is that if a patient is on active therapy, we really have no good surrogate markers to see whether we are achieving that or, if we’re achieving that, whether there might be a benefit to a higher dose intensity. Would we have a benefit of using ruxolitinib at 20 mg twice a day versus 10 mg on the basis of decreasing progression? We don’t have a good way to answer that. Would ruxolitinib and interferon combined be more effective in avoiding progression? Again, we have no good way to prove that at the moment. So, that remains a key unmet need.

Now, what I’m excited about are several things. No. 1: I think the decrease in the heterogeneity of PV care through improved universal guidelines, such as NCCN, is a big advance. And it helps to make us more homogenous in how we’re managing patients, and hopefully that leads to decreased disease suffering and a decrease in thrombotic events. No. 2: I’m excited that there have now been therapies that have been specifically developed for PV. Ruxolitinib is the first therapy that was developed, designed, and carried out with an FDA approval specific in PV. Before that, everything was largely extrapolated from other diseases or other indications.

Finally, there are many additional areas that are being investigated, some in advanced myelofibrosis, but if they prove to be positive, they may well be beneficial in polycythemia vera. And some in polycythemia vera itself, whether it be Nutlin analogs, histone deacetylase inhibition, or other therapies, there remains significant scientific interest as well as interest from the pharmaceutical industry in both identifying unmet needs in patients with polycythemia vera and addressing them therapeutically.

Transcript Edited for Clarity

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