Andrew Kuykendall, MD, of the Moffitt Cancer Center, discusses key findings and implications of a phase II trial that evaluated rusfertide in polycythemia vera (PV).
Dr. Kuykendall and colleagues presented results from the PTG-300-04 trial during the 2023 European Hematology Association Congress.
They hypothesized that rusfertide, a hepcidin mimetic, could decrease erythropoiesis and phlebotomy requirements in PV and alleviate symptoms. It was important address the “area of unmet need” in patients with PV who require periodic therapeutic phlebotomy, Dr. Kuykendall said.
“Unfortunately, therapeutic phlebotomy can be quite challenging for patients, and quite archaic when you think about the idea of draining blood from people as part of a modern approach to hematologic malignancy,” he said.
Dr. Kuykendall pointed to several key issues with regular phlebotomies.
“It’s something that many patients can’t tolerate effectively,” he said. “It really comes with a high cost of having to come into a medical center. You’re tied to the health care system to get bloodwork … it can sometimes take days out of your schedule.”
The fact that “many patients don’t tolerate phlebotomies well” informed the study’s design and its primary endpoint, Dr. Kuykendall
“One of the things that we always primarily wanted to find out is whether or not this is effective in reducing the number of phlebotomies in patients that had previously required them,” he said.
The phase II trial included patients with PV who had at least three phlebotomies with or without concurrent cytoreductive therapy to maintain hematocrit <45% in the 24 weeks before their enrollment.
The study showed that around 60% of patients did not require a therapeutic phlebotomy during 28 weeks of rusfertide treatment, while 26% required one therapeutic phlebotomy during that time period.
Dr. Kuykendall spoke about the implications of the study’s results and what he sees as the next steps.
“It’s very clear, based on what we’ve seen in the phase II data, that rusfertide is able to rapidly and efficiently alleviate the need for phlebotomy in patients that have required phlebotomy previously,” he said. “There is an ongoing phase III study that is that is really going to test that very rigorously.”