6/28/23, 12:25 AM CYTO-PV Pathway Feedback Search Clinical Topics Home Studies CYTO PV CYTO PV Disease Disease Polycythemia Polycythemia vera Trial question What is the role of hematocrit target < 45% in patients with polycythemia vera? Study design Multi-center Open label RCT Population Characteristics of study participants 38.0% female N = 365 62.0% male 365 patients (138 female, 227 male) Inclusion criteria: adults patients with JAK2-positive polycythemia vera who were being treated with phlebotomy, hydroxyurea, or both Key exclusion criteria: hypersensitivity or contraindication to study treatments, significant liver or renal disease, history of active substance or alcohol abuse within the last year, pregnant or lactating women, presence of any life-threatening condition or of any disease that is likely to significantly shorten life expectancy Interventions N=182 low-hematocrit targeted therapy (more aggressive therapy for a hematocrit target of < 45%) N=183 high-hematocrit targeted therapy (less aggressive therapy for a hematocrit target of 45- 50%) Primary outcome https://web.pathway.md/studies/recdR27drgn8VVG2A 1/2 6/28/23, 12:25 AM CYTO-PV Pathway Death from cardiovascular causes or major thrombotic events 9.8 % 9.8 7.4 % 4.9 % Significant decrease 2.7 2.5 % NNT = 14 0.0 % Low-hematocrit targeted therapy High-hematocrit targeted therapy Significant decrease in death from cardiovascular causes or major thrombotic events (2.7% vs. 9.8%; HR 0.26, 95% CI 0.1 to 0.69) Secondary outcomes Significant decrease in total cardiovascular events (4.4% vs. 10.9%; HR 0.37, 95% CI 0.16 to 0.84) Safety outcomes No significant difference in rate of adverse events. Conclusion In adults patients with JAK2-positive polycythemia vera who were being treated with phlebotomy, hydroxyurea, or both, low-hematocrit targeted therapy was superior to high-hematocrit targeted therapy with respect to death from cardiovascular causes or major thrombotic events. Reference Marchioli R, Finazzi G, Specchia G et al. Cardiovascular events and intensity of treatment in polycythemia vera. N Engl J Med. 2013 Jan 3;368(1):22-33. Open reference URL https://web.pathway.md/studies/recdR27drgn8VVG2A 2/2