Trial finds treatment eases PV symptoms despite missing main goal

Early treatment with Jakafi (ruxolitinib) is no better than older therapies at inducing a complete response in those with polycythemia vera (PV), but it may be better at easing certain disease symptoms, according to results from a clinical trial.

The Phase 2 trial, called RuxoBEAT (NCT02577926), was sponsored by RWTH Aachen University in Germany. In the trial, 78 previously untreated adults with PV were randomly assigned to treatment with Jakafi or with best available therapy (BAT). The main goal was to see how many patients achieved a complete response — meaning blood cells were normalized, and patients reported no symptoms.

Results showed the study missed this goal: in patients given either Jakafi or BAT, less than 3% of patients experienced a complete response. But even though the study missed its main goal, secondary outcomes showed benefits with Jakafi relative to BAT.

Full results from the trial were published in Blood Cancer Journal, in the paper “Firstline treatment with ruxolitinib versus best available therapy in patients with polycythemia vera: results from the randomized RuxoBEAT trial.” The work was funded by Novartis, which markets Jakafi.

Recommended Reading

Sept. 3, 2026 by Marisa Horak, MS

Blood cancer treatment advances have saved nearly 26 million life-years

Jakafi outperformed BAT in reducing spleen enlargement

PV is a form of blood cancer marked by the overproduction of certain blood cells, which leads to issues including abnormal blood cell counts and an enlarged spleen. Jakafi is a cytoreductive therapy, meaning a treatment designed to lower blood cell counts. Currently, it is approved as a treatment for PV patients whose disease is not adequately managed after initial treatment with the standard first-line therapy, hydroxyurea.

The RuxoBEAT study was run to evaluate whether Jakafi might offer benefits over hydroxyurea and other first-line options if used as the initial treatment for PV. Although results showed the therapy offered no benefit in terms of complete response rates, secondary outcomes showed that Jakafi was significantly better than BAT at easing patient-reported itching and fatigue.

While [complete response] rates at 6 months were not higher with [Jakafi] than BAT, [Jakafi] was more effective in controlling hematocrit and splenomegaly and alleviating PV-associated pruritus and fatigue.

Jakafi also outperformed BAT in controlling hematocrit (red blood cell levels) and reducing spleen enlargement. However, Jakafi was significantly worse than BAT at controlling production of platelets (cell fragments that help blood clot).

“While [complete response] rates at 6 months were not higher with [Jakafi] than BAT, [Jakafi] was more effective in controlling hematocrit and splenomegaly and alleviating PV-associated pruritus and fatigue,” the researchers concluded.

The researchers noted that this study was limited to a relatively small group of patients — in fact, the trial was originally designed to be larger, but recruitment was ended early due to disruptions related to the COVID-19 pandemic. Overall, the researchers said their results justify further studies to evaluate Jakafi as a potential first-line option in PV.