Future Directions in the Treatment of Polycythemia Vera

Jamile Shammo, MD, FASCP, FACP: We now have options for patients who have polycythemia vera [PV]. I know that other options may be available in the future for patients who have this entity. I think that it’s a good thing that we should be able to offer our patients multiple options. Clearly, patients who have had issues with enlarged spleen aren’t able to tolerate hydroxyurea, and people who had heavy burden with phlebotomy may not be able to tolerate phlebotomy, so ruxolitinib presents a very good option for the treatment of their disease. For patients who are interested in having children in a few months, this may represent a good option for them to achieve that goal. For patients who don’t have that possibility or who had failed all of the above treatments, we will have clinical trials so that we can offer them novel agents that would have the potential for controlling their disease moving forward.

It would be important to see what the clinical trials would bring. I would think that combination therapies may be most helpful in more advanced disease entities like myelofibrosis where you can justify the risk of combination therapy because it’s a much more aggressive disease than it would be the case with PV. Perhaps you could consider that in advanced PV, or maybe you could consider this in patients with PV and multiple mutations. It has to be considered in a certain group of patients. I don’t know that I would necessarily consider combination therapy in all-comers with PV. It has to be thought of in a rational fashion.

We need to understand this group of patients better on a molecular level. We know a lot about the landscape of their molecular profiles, but we know less about how to treat that. We know less about the landscape of people who relapse after a particular treatment and how to address that particular treatment, for example. We probably need to know more about how to utilize transplant in that setting. There’s a lot to be learned about how to use this.

Of course, there is also a whole slew of JAK [Janus kinase] inhibitors that may have various properties as well. It is interesting to think about utilizing them pre- and post-transplant. There are a whole slew of biological agents that could be combined with JAK inhibitors to see how we can make the most out of that combination. We can learn how they can be explored either alone or in combination with stem cell transplantation. There’s a lot that needs to be done, but all that has to be done in the context of a clinical trial. It probably starts with understanding more about the disease biology, taking it to some type of a clinical trial, and keeping our eyes open to get an observation to translate that into some type of conclusion that may be beneficial to patients.

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