Survival and quality-of-life implications of cytopenia trajectories in ruxolitinib-treated myelofibrosis

Abstract

Background: Cytopenia is a common complication in patients with myelofibrosis and may worsen during treatment with ruxolitinib. Methods: The RUX-MF multicenter study evaluated 879 patients treated with ruxolitinib for at least 6 months, categorizing them into four groups based on the evolution of cytopenia: never cytopenic, treatment-emergent cytopenia, persistent cytopenia, and improved anemia. Results: At baseline, 40.6% of patients presented with cytopenia, increasing to 57.8% after 6 months. Baseline cytopenia was associated with significantly reduced median overall survival (OS) compared to noncytopenic patients (3.7 vs. 6.7 years). Prognosis varied notably across groups: patients who remained noncytopenic had the median best OS (8.1 years), whereas those with persistent cytopenia had the worst (3.7 years). Treatment-emergent cytopenia was linked to intermediate outcomes (5.1 years), with isolated thrombocytopenia showing the poorest prognosis (4.3 years) and anemia a slightly better one (6.1 years). Patients with improved anemia had better survival than those with persistent anemia (5.2 vs. 3.5 years). Symptom response mirrored survival trends, with the best outcomes in noncytopenic and improved anemia groups. Conclusions: These findings highlight the prognostic significance of cytopenia dynamics during ruxolitinib therapy and support the use of cytopenia trajectory monitoring as a valuable tool for risk stratification and treatment optimization in myelofibrosis.