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Hepcidin Mimetic Effective in Polycythemia Vera

PTG-300, a hepcidin mimetic, eliminated the need for phlebotomy and the resultant iron deficiency in patients with high- and low-risk polycythemia vera, according to data presented at December’s American Society of Hematology (ASH) virtual meeting.

In this exclusive MedPage Today video, study co-author Andrew Kuykendall, MD, of Moffitt Cancer Center in Tampa, Florida, describes the findings.

Following is a transcript of his remarks:

As a background, polycythemia vera is a chronic leukemia that leads to the overproduction of many blood cells. And what we really worry about in these patients is that they are at increased risk for thrombotic events, meaning, heart attack, strokes, DVTs, blood clots, otherwise. And that really forms the kind of mainstay of our treatment is to try to prevent those thrombotic events as well as maximize quality of life in these chronic leukemias. And the way we do that traditionally has been with the baby aspirin, phlebotomies, to keep the hematocrit less than 45%. And in some cases cytoreductive therapy agents that help to control the blood counts.

So the problem with our current therapy is that oftentimes it requires heavily on phlebotomies to keep the hematocrit below 45%. And that leads to a significant state of iron deficiency, which allows for less need for phlebotomies, but also symptoms that are associated with our inefficiency. And so this trial looks at an agent called PTG-300, which is a hepcidin-mimetic. And the goal of this was to see if we could pharmacologically reduce the need for phlebotomy as well as allowing iron levels to remain in a pretty good zone to avoid those symptoms of iron deficiency. And intriguingly in the small number of patients we’ve treated so far, it appears to do that pretty substantially. And as you can see from this graph the red dots there are where patients were getting phlebotomies, and then once they go on study, those phlebotomies pretty much are eliminated.

And in the abstracts we’re presenting at ASH, we show that the PTG-300 eliminates the needs for phlebotomy, but also is able to regulate these iron levels and seems to be associated with an improvement in symptoms, such as improvement in concentration, fatigue, itching, as well as other symptoms.

  • Greg Laub joined MedPage Today in 2005 as Production Manager and led the launch of the video department in 2007. He is currently responsible for the website’s video production. Follow