Patients with polycythemia vera (PV) are commonly treated with periodic therapeutic phlebotomy (TP), alone or in combination with other agents, to maintain hematocrit levels below 45%. However, TP is associated with increased iron utilization, which leads to suppression of hepcidin, increased iron absorption and recycling, ultimately worsening patients’ iron deficiency and resulting in a continued need for TP. Ronald Hoffman, MD, Icahn School of Medicine at Mount Sinai, New York, NY, shares the findings of the PTG-300-04 Phase II clinical trial (NCT04057040) evaluating the efficacy of rusfertide (PTG-300), a hepcidin mimetic agent, in achieving control of hematocrit levels in patients with PV. The study showed that rusfertide therapy led to the near complete elimination of TP in all subgroups. In addition, hematocrit levels were maintained below 45%. Importantly, patients treated with rusfertide experienced a reversal of systemic iron deficiency and a third of the subjects experienced a reduction of at least 40% in their Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) total symptom scores over the course of the study. There were no drug-related serious adverse events. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.

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