Cardiovascular events and intensity of treatment in polycythemia vera.

Abstract

BACKGROUND Current treatment recommendations for patients with polycythemia vera call for maintaining a hematocrit of less than 45%, but this therapeutic strategy has not been tested in a randomized clinical trial. METHODS We randomly assigned 365 adults with JAK2-positive polycythemia vera who were being treated with phlebotomy, hydroxyurea, or both to receive either more intensive treatment (target hematocrit, <45%) (low-hematocrit group) or less intensive treatment (target hematocrit, 45 to 50%) (high-hematocrit group). The primary composite end point was the time until death from cardiovascular causes or major thrombotic events. The secondary end points were cardiovascular events, cardiovascular hospitalizations, incidence of cancer, progression to myelofibrosis, myelodysplasia or leukemic transformation, and hemorrhage. An intention-to-treat analysis was performed. RESULTS After a median follow-up of 31 months, the primary end point was recorded in 5 of 182 patients in the low-hematocrit group (2.7%) and 18 of 183 patients in the high-hematocrit group (9.8%) (hazard ratio in the high-hematocrit group, 3.91; 95% confidence interval [CI], 1.45 to 10.53; P=0.007). The primary end point plus superficial-vein thrombosis occurred in 4.4% of patients in the low-hematocrit group, as compared with 10.9% in the high-hematocrit group (hazard ratio, 2.69; 95% CI, 1.19 to 6.12; P=0.02). Progression to myelofibrosis, myelodysplasia or leukemic transformation, and bleeding were observed in 6, 2, and 2 patients, respectively, in the low-hematocrit group, as compared with 2, 1, and 5 patients, respectively, in the high-hematocrit group. There was no significant between-group difference in the rate of adverse events. CONCLUSIONS In patients with polycythemia vera, those with a hematocrit target of less than 45% had a significantly lower rate of cardiovascular death and major thrombosis than did those with a hematocrit target of 45 to 50%. (Funded by the Italian Medicines Agency and others; ClinicalTrials.gov number, NCT01645124, and EudraCT number, 2007-006694-91.).

DOI: 10.1056/NEJMoa1208500

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@article{Marchioli2013CardiovascularEA, title={Cardiovascular events and intensity of treatment in polycythemia vera.}, author={Roberto Marchioli and Guido Finazzi and Giorgina Specchia and Rossella Rosaria Cacciola and Riccardo Cavazzina and Daniela Cilloni and Valerio De Stefano and Elena Maria Elli and Alessandra Iurlo and Roberto Latagliata and Francesca Lunghi and Monia Lunghi and Rosa Maria Marfisi and Pellegrino Musto and Arianna Masciulli and Caterina Musolino and Nicola Cascavilla and Giovanni Quarta and Maria Luigia Randi and Davide Rapezzi and Marco Ruggeri and Elisa Rumi and Anna Rita Scortechini and Simone Santini and Marco Scarano and Sergio Siragusa and Antonio Spadea and Alessia Tieghi and Emanuele Angelucci and Giuseppe Visani and Alessandro Maria Vannucchi and Tiziano Barbui}, journal={The New England journal of medicine}, year={2013}, volume={368 1}, pages={22-33} }