Dr Kuykendall on Quality of Life Outcomes With Rusfertide in Polycythemia Vera

“Fortunately, we know our patients with polycythemia vera have a relatively good prognosis. For the most part, if we can avoid major cardiovascular events, the median [overall] survival in [patients with] polycythemia vera is [approximately] 15 to 20 years.”

Andrew Kuykendall, MD, an assistant member in the Department of Malignant Hematology at the Moffitt Cancer Center, detailed the role rusfertide (PTG-300) plays in patients with polycythemia vera and its effect on their quality of life (QOL).

Patients with polycythemia vera typically have a relatively good prognosis if major cardiovascular events can be avoided, Kuykendall began. He noted that the median survival is approximately 15 to 20 years; therefore, patients could live with the disease for years. However, the disease could inevitably affect patients’ QOL, he stated. Specifically, patients could experience chronic and vague symptoms of fatigue, brain fog, and decreased attentiveness, he explained. However, with the mechanism of action of rusfertide and by targeting the iron pathway, it could help alleviate iron deficiency and improve QOL, he added.

Furthermore, although it’s important to focus on modifying and eliminating the disease, it’s also just as important to focus on patients’ QOL, especially for those with chronic diseases, Kuykendall emphasized. When evaluating the efficacy and safety of an agent, it’s also essential to build upon patient-reported outcomes, he continued. In the phase 3 VERIFY study (NCT05210790), assessing the PROMIS fatigue score helped identify the effect fatigue has on patients, he explained. Of note, patients treated with rusfertide demonstrated a dramatic improvement in their levels of fatigue compared with placebo, according to Kuykendall. Moreover, results using the myelofibrosis symptom assessment form revealed that patients showed improvement in their total symptom score from 0 to 32 on rusfertide vs placebo, he asserted. Therefore, rusfertide has demonstrated the ability to control hematocrit, decrease the necessity for phlebotomy, and improve the QOL in patients with polycythemia vera, he concluded.