Prospective, non-interventional study of ruxolitinib therapy in patients with polycythaemia vera - German real-world data (PaVe study).

Ruxolitinib, a JAK1/JAK2 inhibitor, has demonstrated efficacy in achieving hematocrit control, spleen volume reductions, and symptom improvement in patients with polycythaemia vera (PV) resistant to or intolerant of hydroxyurea in clinical trials. PaVe is a prospective, noninterventional study of patients with PV who received ruxolitinib in a real-world setting in Germany. Observational parameters included effectiveness, safety, patient satisfaction and quality of life. Patients with and without prior treatment with ruxolitinib were included in this study. Additionally, patients were stratified by no/high risk. The study was primarily analyzed using descriptive methods. Four hundred and thirty-three patients with PV were included in this study, of which 62% were pretreated with a JAK-inhibitor and 38% were not. Mean decreases from Baseline in hematocrit levels/leukocyte counts at the last assessment were more pronounced in naïve patients (-6.2% versus - 1.6%/-4.5 × 103/µl versus - 1.4 × 103/µl) and high-risk patients (-6.3% versus - 5.3%/-4.8 × 103/µl versus - 2.6 × 103/µl). Mean decreases from Baseline in thrombocyte counts at the last assessment were more pronounced in naïve patients (-80.5 × 103/µl versus - 42.6 × 103/µl) and no-risk patients (-108.0 × 103/µl versus - 77.1 × 103/µl). There was almost no difference in the total number of thromboembolic events in JAK inhibitor naïve versus JAK inhibitor pretreated patients (3.1%, n = 5 versus 3.8%, n = 10). No new safety concerns were observed with ruxolitinib. Findings from this study in a real-world setting show that ruxolitinib is an effective and safe second-line treatment in PV patients.