Rusfertide May Reduce Need for Therapeutic Phlebotomy in Polycythemia Vera


Yelena Ginzburg, MD, discusses a long-term goal of the use of rusfertide in patients with chronic polycythemia vera.

Yelena Ginzburg, MD, associate professor of medicine, hematology, and medical oncology at Mount Sinai, discusses a long-term goal of the use of rusfertide (PTG-300) in patients with chronic polycythemia vera.

Polycythemia vera is one of several types of myeloproliferative neoplasms; it is often treated with therapeutic phlebotomy. In the phase 2 PTG-300-08 trial (NCT04767802), patients received rusfertide to evaluate whether twice-weekly treatment followed by weekly maintenance doses could maintain hematocrit levels below 45% without phlebotomy. Results presented at the 2021 American Society of Hematology annual meeting showed that all 16 patients who received rusfertide had reduced and maintained lower hematocrit levels.

According to Ginzburg, a long-term goal of the use of rusfertide is a prolonged reduction of the need for phlebotomy, which could also lead to a reduced risk of thrombosis. Though this was not an end point for this study, further research with rusfertide could confirm that patients with polycythemia vera have fewer phlebotomy procedures in the long term and fewer thrombotic events.

The phase 3 VERIFY study (NCT05210790) will evaluate the efficacy of rusfertide versus placebo in a larger patient population and include the mean number of phlebotomies required by patients as a secondary end point.


0:08 | I think the main point that we are hoping to see with the trial as it continues is both a prolonged, continuous either phlebotomy-free or phlebotomy-reducing effect of using rusfertide in the chronic setting that can also ultimately allow us to evaluate a potential reduction in the thrombotic events in this patient population. So, if the patients who require high levels of phlebotomy have a higher risk of thrombosis in this PV population, then reducing the need for therapeutic phlebotomy may uncover a significant reduction in the risk of thrombotic events in this patient population. Although that requires a longer time for evaluating and it is not really an end point that is required, we are hopeful that—not in this trial—but the use of this agent will eventually increase that additional benefit for this patient population.

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