Ruxolitinib Treatment Effective, Safe for Patients With Newly Diagnosed Polycythemia Vera

Futility Analysis of the Phase 2b RuxoBEAT Clinical Trial 

Ruxolitinib therapy for patients with untreated polycythemia vera (PV) was found to be feasible, well-tolerated, and efficient, according to a futility analysis of the phase 2b RuxoBEAT clinical trial published in the Annals of Hematology. 

Patients with polycythemia vera, a myeloproliferative neoplasm, “suffer from pruritus, night sweats, and other symptoms, as well as from thromboembolic complications and progression to post-PV myelofibrosis,” wrote Steffen Koschmieder, MD, RWTH Aachen University, Pauwelsstr, Aachen, Germany, and colleagues. Ruxolitinib is approved for second-line therapy in high-risk PV pts with hydroxyurea intolerance or resistance. 

The RuxoBEAT trial is a multicenter, open-label, 2-arm, phase 2b trial with a target population of 380 pts with PV or essential thrombocythemia (ET), randomized to receive ruxolitinib or the best available therapy. The pre-specified futility analysis of the trial analyzed the clinical benefit and tolerability of ruxolitinib among previously untreated patients with PV. 6-week cytoreduction was allowed.

At the time of the futility analysis, there were 28 patients who received ruxolitinib for at least 6 months. Compared to baseline, after 6 months of treatment, there was a significant reduction of median hematocrit (46% to 41%), the median number of phlebotomies per year (4.0 to 0), and median patient-reported pruritus scores (2 to 1), and a trend toward a reduction in night sweat scores (1.5 to 0). There were 109 adverse events, all grades 1 to 3, occurred in 24 out of 28 patients. No patient permanently discontinued treatment because of adverse events. 

“The futility analysis of the RuxoBEAT trial confirms that treatment with [ruxolitinib] in untreated PV patients is effective (regarding the above-mentioned endpoints), feasible, and well-tolerated,” Dr Koschmieder and co-authors concluded. The trial is currently ongoing.

Source:

Koschmieder S, Isfort S, Wolf D, et al. Efficacy and safety of ruxolitinib in patients with newly-diagnosed polycythemia vera: Futility analysis of the RuxoBEAT clinical trial of the GSG-MPN study group. Ann Hematol. 2023;102(2):349-358. doi:10.1007/s00277-022-05080-7