Heterogeneity within AML with CEBPA mutations; Only CEBPA double mutations, but not single CEBPA mutations are associated with favourable prognosis

Abstract

CCAAT/enhancer binding protein alpha (CEBPA) mutations in AML are associated with favourable prognosis and are divided into N- and C-terminal mutations. The majority of AML patients have both types of mutations. We assessed the prognostic significance of single (n7) and double (n12) CEBPA mutations among 224 AML patients. Double CEBPA mutations conferred a decisively favourable overall (P0.006) and disease-free survival (P0.013). However, clinical outcome of patients with single CEBPA mutations was not different from CEBPA wild-type patients. In a multivariable analysis, only double - but not single - CEBPA mutations were identified as independent prognostic factors. These findings indicate heterogeneity within AML patients with CEBPA mutations. © 2009 Cancer Research UK.