Srdan Verstovsek, MD

Srdan Verstovsek, MD, PhD, a professor of Medicine and a hematologic oncologist at the University of Texas MD Anderson Cancer Center, discusses the main reasons for his study on the use of rusfertide (PTG-300) in patients with polycythemia vera (PV).

Patients with PV are often faced with severe fatigue due to an increase in iron utilization by the disease. After repeated therapeutic phlebotomy, iron deficiency only worsens. Rusfertide has helped this by achieving hematocrit control while eliminating the need for therapeutic phlebotomy as well as improving the quality of life in patients with PV due to elimination of the higher red blood cell count which can affect iron levels.

Findings from a phase 2 trial concluded rusfertide to be both well tolerated and effective in achieving the target hematocrit (HCT) of < 45% without phlebotomy when induction therapy was administered twice a week.The phase 3 trial which looks to confirm the previous phase 2 results and assess the primary end points of a confirmed HCT of 45% or less.

 | The key point would be to eliminate the need for phlebotomy. That is the value that we understand is the number one goal in polycythemia vera patients. But along the way, because other issues may arise in the life of the policy team of our patients, we want to know and make sure that the therapy has other benefits. And you may understand that the quality of life is the paramount. You want to have a therapy that not only normalizes the blood numbers, but that improves the quality of life. So we're going to look at the two particular aspects of the benefit: normalization and elimination of phlebotomy.

 | Normalization of the hematocrit below 45% and elimination of phlebotomy, durability of that benefit, making sure that there are no ups and downs, that this therapy works all the time, that the therapies given in a safe way, is given by the patients, so durability is the another key secondary endpoint, but also improves the quality of life measured by different questionnaires. You want to objectivize that benefit on quality of life, by looking at specific issues that polycythemia vera patients suffer from. And finally, if these issues of quality of life are related to the iron levels, we're going to, of course, measure ferritin and make sure that these parameters correlate with improvements in quality of life, and prove the point that normalization of iron elevation of iron deficiency is beneficial for the patients.

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Srdan Verstovsek, MD, PhD, discusses the main reasons for his study on the use of rusfertide in patients with polycythemia vera.

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Srdan Verstovsek, MD | Authors

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