Dr Kuykendall on Clinical Benefit With Rusfertide in Phlebotomy-Dependent Polycythemia Vera

"With rusfertide, the mean number of phlebotomies over the 32 weeks on the study actually went down to 0.5, drastically reducing the phlebotomy requirements of these patients who had previously been receiving consistent phlebotomies before going on study."

Andrew Kuykendall, MD, assistant member in the Department of Malignant Hematology at Moffitt Cancer Center, shared findings from the phase 3 VERIFY trial (NCT05210790) evaluating the investigational hepcidin mimetic peptide rusfertide (PTG-300) vs placebo in phlebotomy-dependent patients with polycythemia vera receiving standard-of-care therapy.

Therapeutic phlebotomies are a cornerstone of polycythemia vera management and are used to maintain hematocrit levels consistently below 45% to reduce thrombotic risk, Kuykendall began by explaining. He noted that many patients require frequent phlebotomies, often every 1 to 3 months. The VERIFY trial focused on patients who were receiving a significant number of phlebotomies—defined as at least 3 in the prior 6 months or at least 5 in the previous year, Kuykendall detailed.

Findings presented at the 2025 ASCO Annual Meeting showed that rusfertide administration substantially reduced the phlebotomy burden, with the mean number of phlebotomies over the 32-week study period decreasing from 1.8 to 0.5. Kuykendall emphasized that this represented a marked reduction in the need for phlebotomies compared with baseline.

An analysis of patient-reported outcomes was also conducted in this trial and showed that patients treated with rusfertide exhibited more pronounced improvements in fatigue compared with those receiving placebo, Kuykendall stated. Improvements in total symptom scores as assessed by the Myelofibrosis Symptom Assessment Form, a validated tool used in myeloproliferative neoplasms, were also reported. Patients receiving rusfertide experienced greater reductions in symptom burden from baseline compared with placebo, he noted.

Overall, rusfertide not only effectively controlled hematocrit and reduced the need for phlebotomies but also demonstrated potential to improve quality of life in patients with polycythemia vera, Kuykendall concluded.