{Reference Type}: Observational Study
{Title}: Ruxolitinib in patients with polycythemia vera resistant and/or intolerant to hydroxyurea: European observational study.
{Author}: Theocharides A;Gisslinger H;De Stefano V;Accurso V;Iurlo A;Devos T;Egyed M;Lippert E;Delgado RG;Cantoni N;Dahm AEA;Sotiropoulos D;Houtsma E;Smyth A;Iqbal A;Di Matteo P;Zuurman M;Te Boekhorst PAW;
{Journal}: Eur J Haematol
{Volume}: 112
{Issue}: 3
{Year}: 2024 Mar 30
{Factor}: 3.674
{DOI}: 10.1111/ejh.14124
{Abstract}: <B>BACKGROUND: </B>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.<BR><B>METHODS: </B>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.<BR><B>RESULTS: </B>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.<BR><B>CONCLUSIONS: </B>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.