What are the treatment options for hydroxyurea resistant or refractory PV?

FAQ published on December 16, 2020
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Raajit K. Rampal, MD, PhD
Assistant Member
Clinical Director, Leukemia Service
Memorial Sloan Kettering Cancer Center
New York, New York
What are the treatment options for hydroxyurea resistant or refractory PV?

Hi, I’m Dr. Raajit Rampal and I’m frequently asked, what are the treatment options for hydroxyurea resistant or refractory polycythemia vera? The definition of resistant or refractory polycythemia vera really centers around the patient having side effects from hydroxyurea, such as fevers or ulcers or mucocutaneous injury, as well as having cytopenias develop while on hydroxyurea at doses that are required to maintain the patient’s hematocrit under 45%. As well, if one is using 2 grams of hydroxyurea a day or more in order to achieve a hematocrit control but still having leukocytosis, thrombocytosis, or splenomegaly that isn’t improving, those all fit the definition of refractory resistant polycythemia vera. In the patient who is on hydroxyurea, switching to interferon represents a reasonable choice. The other choice is switching to the JAK1/2 inhibitor ruxolitinib. This was based on the RESPONSE trial in which patients were randomized to ruxolitinib or standard therapy who met the definition of resistant or refractory PV while on hydroxyurea, and in fact this data showed that spleen size and hematocrit were well-controlled and better controlled in patients who received ruxolitinib than who had standard therapy, and that includes hydroxyurea. As well, with symptom burden improved significantly in patients who were treated with ruxolitinib, so this is a standard of care option that one can consider. It’s also always important to consider investigational options for these patients. Thank you for viewing this activity.

Last modified: December 8, 2020