ArticleBlood advances2026
A dynamic prognostic model for polycythemia vera long-term outcomes in patients treated with ruxolitinib.
Article in Blood advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06134102 (Clinical and Laboratory Characteristics of Polycythemia Vera), which is not on this map. Not yet cited in PubMed.
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Clinical and Laboratory Characteristics of Polycythemia Vera
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Abstract
abstractRuxolitinib is widely used in polycythemia vera (PV) following hydroxyurea failure. However, validated response criteria to predict long-term outcomes in ruxolitinib-treated patients are lacking, complicating clinical decision-making. We investigated predictors of event-free survival ([EFS]; including progression to post-PV myelofibrosis, thrombosis, hemorrhages, or death) after 6 months of ruxolitinib in 178 patients with PV enrolled in the observational PV-ARC study. After a median follow-up from ruxolitinib start of 3.50 years, 15 patients died, 7 had a thrombosis, 9 had a hemorrhage, and 21 progressed to myelofibrosis. Overall, 5-year EFS was 70.4%. Leukocytosis, thrombocytosis, phlebotomy need, lack of spleen length reduction ≥50% (SR50), and ruxolitinib dose <10 mg twice daily at 3 time points (baseline and months 3 and 6), were tested for association with EFS. Multivariable analysis identified 3 independent risk factors: (1) ruxolitinib dose <10 mg twice daily at ≥1 time point (hazard ratio [HR], 1.94; P = .047), (2) no SR50 at months 3 and 6 (HR, 2.65; P = .009), (3) phlebotomy requirement at ≥2 time points (HR, 2.11; P = .039). Points were assigned as follows: 1 to phlebotomies at 1 time point; 2 to phlebotomies at ≥2 time points and to ruxolitinib <10 mg twice daily at ≥1 time point; and 2.5 to lack of SR50. Based on cumulative scores, we developed the PV-response to ruxolitinib after 6 months (PV-RR6) prognostic model, identifying 3 risk categories: low (score 0: 5-year EFS, 89.4% [n = 63]), intermediate (score 1-2.5: EFS, 71.0% [n = 82]), and high (score >2.5: EFS, 38.9% [n = 33]). PV-RR6 enables early identification of patients at risk of poor outcomes, supporting timely treatment optimization in ruxolitinib-treated patients. This trial was registered at www.clinicaltrials.gov as NCT06134102.
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