ERNEST-2 retrospective validation: Prognostic value of anemia + thrombocytopenia in primary MF

Results from a retrospective validation study assessing the prognostic value of anemia and thrombocytopenia for patients with overt primary myelofibrosis (PMF), were published in Leukemia by Barosi et al. Outcomes of 559 patients from the ERNEST-2 registry were compared with a Pavia-Centre for the Study of Myelofibrosis (CSM) database validation cohort of 530 adults with overt PMF.  

Key data: In ERNEST-2, patients with anemia + thrombocytopenia had the shortest median overall survival (OS; 1.7 years; interquartile range [IQR], 0.7–4.4), whereas in the Pavia cohort the lowest median OS was observed with severe anemia only (5.4 years; IQR, 2.2–11.7). At 5 and 10 years, the cumulative incidence of death in the ERNEST-2 cohort was 57% and 86% in subjects with severe anemia alone and 77.9% and 85.9% in those with anemia + thrombocytopenia, while in the Pavia cohort it was 44.8% and 71.2% with severe anemia alone and 45.3% and 76.8% with anemia + thrombocytopenia. Receiver operating characteristic (ROC) curve analysis showed that anemia + thrombocytopenia had poor predictive value for 10-year survival in both cohorts, with area under the curve (AUC) values of 0.52 in ERNEST-2 and 0.50 in the Pavia cohort. 

Key learning: Anemia + thrombocytopenia demonstrated limited prognostic discrimination across cohorts, emphasizing the need for hybrid models that integrate clinical and molecular features to improve survival prediction in overt PMF.