Ruxolitinib was superior to standard therapy in patients intolerant to or inadequately responded to hydroxyurea for the treatment of polycythemia vera, a study published in the New England Journal of Medicine has shown.
or the phase 3, researchers sought to investigate the safety and efficacy of ruxolitinib, a Janus kinase (JAK) 1 and 2 inhibitor, as second-line treatment for polycythemia vera. Researchers enrolled 222 phlebotomy-dependent patients with splenomegaly and randomly assigned them 1:1 to receive either ruxolitinib or standard therapy.
Results showed that 21% of those in the ruxolitinib group achieved hematocrit control through week 32 and had at least a 35% decrease in spleen volume at week 32 compared with only 1% of patients in the standard therapy group (P < 0.001).
In addition, 60% and 20% of those in the ruxolitinib and standard therapy groups, respectively, achieved hematocrit control, while 38% of the ruxolitinib group versus 1% of the standard therapy group had a 35% decrease in spleen volume.
Furthermore, 24% of ruxolitinib patients achieved a complete hematologic remission versus 9% of standard-therapy patients (P = 0.003). In regard to safety in the ruxolitinib group, 2% of patients experienced grade 3 or 4 anemia, 5% had grade 3 or 4 thrombocytopenia, and 6% developed a herpes zoster infection.
Based on these study results, the U.S. Food and Drug Administration approved ruxolitinib for the treatment of patients with polycythemia vera in Decemeber 2014, making it the first drug approved by the FDA for this condition.
Ruxolitinib, a Janus kinase (JAK) 1 and 2 inhibitor, was shown to have a clinical benefit in patients with polycythemia vera in a phase 2 study. Authors conducted a phase 3 open-label study to evaluate the efficacy and safety of ruxolitinib versus standard therapy in patients with polycythemia vera who had an inadequate response to or had unacceptable side effects from hydroxyurea.