Prior cytopenia predicts worse clinical outcome in acute myeloid leukemia

• AML patients with and without antecedent cytopenias were compared.
• AML patients with prior cytopenias were older and more likely to have dysplasia.
• Prior thrombocytopenia independently predicts prognosis in multivariable analysis.

The prognosis of acute myeloid leukemia (AML) is influenced by both disease-intrinsic and patient-related factors. In particular, AML following myelodysplastic syndrome (MDS) (AML with myelodysplasia-related changes, AML-MRC) has a poor prognosis. We hypothesized that patients with cytopenias prior to AML, but no known prior MDS, may share biologic features with AML-MRC. We evaluated 140 AML patients without prior MDS who had complete blood count (CBC) data available 6–36 months prior to their diagnosis. Cytopenia, defined as clinically unexplained thrombocytopenia or macrocytic anemia, was present in 29/140 (21%) patients. Compared to non-cytopenic patients, AML patients with prior cytopenia were older and more often met morphologic or cytogenetic criteria for AML-MRC. Prior cytopenia was associated with shorter survival in patients with intermediate-risk cytogenetics (median OS 4.2 versus 24.1 months, p < 0.0001), but not in patients with adverse-risk cytogenetics (median OS 4.4 versus 6.0 months, p = 0.57). Prior thrombocytopenia, but not macrocytic anemia, was significantly associated with shorter overall survival (p = 0.01) independent of treatment approach, karyotype risk, and age on multivariable analysis. Our data suggest that AML patients with prior cytopenias have features similar to AML-MRC, and in particular support the use of prior unexplained thrombocytopenia as an independent marker of high-risk disease.