Ruxolitinib versus best available therapy in inadequately controlled polycythemia vera without splenomegaly: a 5-year results from the phase 3, randomized, RESPONSE-2 study
BACKGROUND AND METHODS
Polycythemia vera (PV) is a myeloproliferative neoplasm for which long-term therapy is required, meaning there is a need for therapies which are not only effective but are also well tolerated over prolonged periods of time. HU is the most used first-line treatment for PV; however, a considerable proportion of patients are intolerant or become resistant. The RESPONSE-2 study (NCT02038036) was a randomized, open-label, multicenter phase 3b study that evaluated the long-term efficacy and safety results of ruxolitinib versus BAT in PV patients with HU-resistance or -intolerance without splenomegaly. The primary analysis of RESPONSE-2 has been previously published and demonstrated superiority of ruxolitinib over BAT for Hct control (62% versus 19%; odds ratio 7.28; P<0.0001), normalizing blood cell counts, and improving PV symptoms after 28 weeks. Here, long-term efficacy and safety results of the RESPONSE-2 study after 260 weeks are reported.
1. Efficacy Mehr