Ruxolitinib in patients with polycythemia vera resistant and/or intolerant to hydroxyurea: European observational study

Background Hydroxyurea (HU) is a commonly used first-line treatment in patients with polycythemia vera (PV). However, approximately 15%-24% of PV patients report intolerance and resistance to HU.Methods This phase IV, European, real-world, observational study assessed the efficacy and safety of ruxolitinib in PV patients who were resistant and/or intolerant to HU, with a 24-month follow-up. The primary objective was to describe the profile and disease burden of PV patients.Results In the 350 enrolled patients, 70% were >60 years old. Most patients (59.4%) had received >= 1 phlebotomy in the 12 months prior to the first dose of ruxolitinib. Overall, 68.2% of patients achieved hematocrit control with 92.3% patients having hematocrit <45% and 35.4% achieved hematologic remission at month 24. 85.1% of patients had no phlebotomies during the study. Treatment-related adverse events were reported in 54.3% of patients and the most common event was anemia (22.6%). Of the 10 reported deaths, two were suspected to be study drug-related.Conclusion This study demonstrates that ruxolitinib treatment in PV maintains durable hematocrit control with a decrease in the number of phlebotomies in the majority of patients and was generally well tolerated.

Automated summary

This study demonstrates that ruxolitinib treatment in patients with polycythemia vera who are resistant and/or intolerant to hydroxyurea maintains durable hematocrit control, reduces the need for phlebotomies, and is generally well tolerated.