Long-term safety and effectiveness of ruxolitinib in Japanese patients with polycythemia vera: a post-marketing study.

AIM: This study evaluated the long-term safety and effectiveness of ruxolitinib in Japanese patients with polycythemia vera in clinical practice.

PATIENTS AND

METHODS

This multicenter, single-arm, non-interventional, prospective, observational study assessed patients receiving ruxolitinib after approval.

RESULTS

Of 550 patients (median age: 71 years, mean disease duration: 5.43 years, median treatment duration: 1091 days), 51.27% reported adverse drug reactions (ADRs) and 12.18% had serious ADRs, with anemia being the most common ADR. The highest incidence of ADRs (40%) was observed from treatment initiation to Day 182, with no tendency for increased incidence thereafter. ADRs of special interest included myelosuppression (27.64%), hepatic impairment (13.45%), infections (9.27%), hemorrhagic events (5.64%), cardiac failure (1.27%), malignancy (1.09%), interstitial lung disease (0.36%), and tuberculosis (0.18%). Baseline hepatic impairment did not increase the incidence of ADRs, but numerical incidence was higher in patients with baseline renal impairment, with similar events being observed in patients with or without renal impairment. At 36 months, spleen response and symptom improvement were observed in 31.43% and 61.14% of patients, respectively. Reductions in hematocrit, white blood cell count, and platelet count were observed throughout the treatment duration.

CONCLUSION

Ruxolitinib demonstrated sustained effectiveness with no new safety concerns in the real-world setting.