A new comparison study showed that among polycythemia vera patients who were resistant or intolerant to hydroxyurea, those treated with ruxolitinib (Jakavi) had a significantly reduced risk of thrombosis and death compared to those who received best available therapy. The study findings are based on a comparison of patients in the phase III RESPONSE clinical trial and the real-world Spanish Grupo Español de Enfermedades Mieloproliferativas Crónicas Filadelfia Negativas (GEMFIN) patient registry. The new findings were presented by Alvarez-Larran et al at the 23rd Annual Congress of the European Hematology Association (EHA) (Abstract PF628).
“When you can complement clinical trial data with real-world experiences, it can provide valuable insight into how treatments affect patients in their day-to-day lives,” said lead study investigator Alberto Alvarez-Larran, MD, of the Hematology Department, Hospital Clinic, Barcelona. “This latest research supports the use of ruxolitinib to help people with polycythemia vera gain better control of their disease when hydroxyurea is not an option.”
The current study compared overall survival and thrombosis rates using data from patients treated in the ruxolitinib arm of the previously reported RESPONSE trial and patients treated in a real-world setting with best available therapy from the GEMFIN registry.
In the RESPONSE trial, the high rate of crossover from best available therapy to ruxolitinib precluded the comparison of overall survival and thrombosis rates. RESPONSE was a global, open-label study that included patients with polycythemia vera resistant or intolerant to hydroxyurea, who were randomly assigned 1:1 to receive either ruxolitinib (at a starting dose of 10 mg twice daily) or best available therapy, which was defined as investigator-selected monotherapy or observation only.
The GEMFIN registry patients in the real-world best-available-therapy group had resistance or intolerance to hydroxyurea according to modified European Leukemia Net criteria and received hydroxyurea (44%), busulfan (10%), radioactive phosphorus (2%), interferon (6%), anagrelide (12%), other therapy (11%), or no cytoreductive therapy (26%). Some patients were treated with multiple therapies.
In the GEMFIN study, patients treated with ruxolitinib had a significantly prolonged overall survival (hazard ratio [HR] = 0.28, 95% confidence interval [CI] = 0.11–0.72) and a lower risk of blood clots (HR = 0.21, 95% CI = 0.06–0.76]) compared to real-world patients treated with best available therapy.
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