Patients with polycythemia vera who are treated with rusfertide may not have to get a blood draw as frequently, research found.
The novel drug, rusfertide, may help mitigate the overproduction of red blood cells — a process called erythrocytosis — in patients with polycythemia vera, a type of myeloproliferative neoplasm (MPN), leading to a less frequent need for blood draws, according to data from the REVIVE trial published in The New England Journal of Medicine.
The study was broken into two parts: part 1 included 70 patients and part 2 included 59 patients — 30 of whom were assigned to receive rusfertide, while the other 29 received a placebo for a period of 12 weeks.
The main goal of the study was response, defined by the percentage of red blood cells in the blood, absence of the need for blood draws and the completion of phase 2 of the trial. Additionally, patient-reported outcomes measuring symptoms were assessed on a score of 0 to 10.
Before the study started, patients underwent an average of 8.7 phlebotomies (blood drawn via a needle in the vein) per year. Then, during part 1 of the study, the average number of phlebotomies dropped to 0.6.
The average maximum hematocrit — which, according to the Red Cross is the percentage, by volume, of red blood cells in the blood — was 44.5% during part 1 of the trial, while it was 50% during the 28 weeks before receiving the first dose of the drug.
“Rusfertide shows great promise for achieving sustained hematocrit control in polycythemia vera patients. Just as importantly, it decreased the need for repeat phlebotomies, with some patients remaining virtually free of the procedure for more than two and a half years,” study author Dr. Marina Kremyanskaya, an associate professor of Medicine (Hematology and Medical Oncology) at the Icahn Mount Sinai, said in a press release about the findings.
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Between the start of therapy and the end of part 1, rusfertide was associated with a decrease in symptom scores in the MPN-SAF in patients who were experiencing moderate or severe symptoms at baseline. According to the National Institutes of Health, the MPS-SAF is a questionnaire that has nine items measuring common symptoms that come with an MPN diagnosis (concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss and fever).
Additionally, in part 2 of the study, 60% of patients responded to rusfertide, compared with 17% who were given the placebo.
“Rusfertide appears to represent a significant step forward in treating polycythemia vera through its unique approach of limiting the amount of iron available for blood cell production,” Kremyanskaya said.
According to the press release, the drug works by mimicking hepcidin, which is a hormone that the liver produces to regulate iron trafficking. By blocking the export of iron to the blood, resfertide creates an iron deficiency and decreases the production of red blood cells. Of note, the drug is given via injection, and patients can administer it to themselves at home.
Throughout the whole study, 13% of patients experienced a moderate (grade 3) side effect, with no patients experiencing a severe (grade 4) or fatal (grade 5) side effect. Most commonly, patients experienced reactions at the injection site.
Now, a phase 3 clinical trial is underway in cancer treatment centers around the globe.
“Pending further clinical studies, this injectable agent could become a valuable therapeutic tool for a disease which many patients and their physicians struggle to bring under control,” Kremyanskaya said.
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