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Real-world ERNEST-2 registry study: Clinical characteristics and outcomes in patients with overt primary MF and cytopenias
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Overt PMF can present with uni-lineage or multi‐lineage cytosis and isolated or multiple cytopenias; the latter known as a cytopenic phenotype (CyP).1 Patients harboring a CyP have worse prognoses and more limited treatment options than those with other types of MF.1 To examine the representation, reliability, and comparability of real‐world data, Guglielmelli et al.1 compared the clinical characteristics and outcomes of patients with MF and a CyP (n = 275; defined by the presence of ≥1 cytopenia at diagnosis) or myeloproliferative phenotype (MyP) (n = 284) in the prospective real-world ERNEST-2 registry cohort study. Results were published in Hemasphere.1 |
| Key learnings |
| Patients with CyP were more likely to be older (p < 0.001), have higher IPSS risk categories (Int2/High, 70.8% vs 29.3%; p < 0.001), reduced JAK2V617F-mutation frequency (58.4% vs 71.9%; p = 0.004), and fewer thrombotic events (12.4% vs 18.3%; p = 0.051) than those with MyP. |
| Death was reported in 80.7% of patients with CyP vs 59.9% of those with MyP (p < 0.001). Patients with anemia and thrombocytopenia had the highest incidence of death (26.8% pt-y; 95% CI, 20.0–35.9; p = 0.003). |
| Poor survival outcomes were associated with increased age (HR, 1.06; 95% CI, 1.05–1.07; p < 0.001), male sex (HR, 1.63; 95% CI, 1.25–2.13; p < 0.001), ≥1% PB (HR, 2.18; 95% CI, 1.56–3.05; p < 0.001), systemic symptoms (HR, 1.34; 95% CI, 1.05–1.72; p = 0.019), and leukocyte count ≥20 x 109/L (HR, 1.87; 95% CI, 1.28–2.72; p < 0.001) in both cohorts. |
| In the CyP cohort, moderate anemia (HR, 1.59; 95% CI, 1.19–2.15; p = 0.002), severe anemia (HR, 1.82; 95% CI, 1.21–2.72; p = 0.004), thrombocytopenia (HR, 2.44; 95% CI, 1.36–4.36; p = 0.003), and anemia + thrombocytopenia (HR, 3.62; 95% CI, 2.20–4.82; p < 0.001) were associated with increased risk of death. |
| The risk of BP transformation was higher in the CyP cohort compared with the MyP cohort (16.4% vs 10.6%; p < 0.0001). |
| This study represents the largest prospective analysis of cytopenic patients and provides a thorough evaluation of clinical correlates and outcomes. Findings confirm that patients with PMF harboring a CyP had poorer prognoses and higher mortality rates compared to those with a MyP. |
Abbreviations: BP, blast phase; CI, confidence interval; CyP, cytopenic phenotype; ESRNEST-2, European Research Network on MPN study; HR, hazard ratio; Int2, intermediate 2; IPSS, International Prognostic Scoring System; JAK, Janus Kinase; MF, myelofibrosis; MyP, myeloproliferative phenotype; PB, peripheral blast; PMF, primary myelofibrosis; pt-y, patient-years.
- Guglielmelli P, Ghirardi A, Carobbio A, et al. Cytopenic overt primary myelofibrosis at presentation: Analysis of outcomes in the prospective, real-world ERNEST-2 registry. Hemasphere. 2025;9(2):e70072. DOI: 10.1002/hem3.70072.
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