Dynamic Model for Predicting Death Within 12 Months in Patients With Primary or Post–Polycythemia Vera/Essential Thrombocythemia Myelofibrosis

Abstract

Purpose Current prognostic tools in myelofibrosis (MF) fail to identify patients at the highest risk of death and are limited by their applicability only to the time of diagnosis. We aimed to define an accelerated phase (AP) in MF by characterizing disease features that can identify patients with median overall survival of ≤ 12 months at any time in the disease course. Patients and Methods Baseline characteristics of 370 consecutive patients with MF from a single center were analyzed to identify features associated with a median overall survival of ≤ 12 months. These putative AP features were then validated by following the course of chronic-phase patients (no AP features at baseline) until the development of one or more AP features and determining their subsequent survival. Results The following three characteristics were associated with poor survival at baseline and were selected as putative AP features: blasts in blood or bone marrow ≥ 10%, platelets less than 50 × 109/L, and chromosome 17 aberrations (median overall survival, 10, 12, and 5 months, respectively). In the validation phase, chronic-phase patients who developed AP features during follow-up were found to have short subsequent survival times (median overall survival, 12, 15, and 6 months, respectively). AP was a necessary step in the progression to blast phase, with leukemic transformation being exceedingly rare (3% risk at 10 years) in patients who remained persistently in chronic phase. Conclusion Blood or bone marrow blasts ≥ 10%, platelets less than 50 × 109/L, and chromosome 17 aberrations defined AP in patients with MF. Patients in AP should be candidates for intensive therapeutic interventions.