Long-term follow-up of cytogenetically normal CEBPA-mutated AML

The aim of this study was to analyze the long-term survival of AML patients with CEBPA mutations. We investigated 88 AML patients with a median age of 61 years and (1) cytogenetically normal AML (CN-AML), (2) monoallelic (mo CEBPA ) or biallelic (bi CEBPA ) CEBPA mutation, and (3) intensive induction treatment. 60/88 patients have been described previously with a shorter follow-up. Median follow-up time was 9.8 years (95% CI: 9.4-10.1 years) compared to 3.2 and 5.2 years in our former analyses. Patients with bi CEBPA mutations survived significantly longer compared to those with mo CEBPA (median overall survival (OS) 9.6 years vs. 1.7 years, p = 0.008) . Patients ≤60 years and bi CEBPA mutations showed a favorable prognosis with a 10-year OS rate of 81%. Both, bi- and mo CEBPA- mutated groups had a low early death (d60) rate of 7% and 9%, respectively. Complete remission (CR) rates for bi CEBPA - and mo CEBPA- mutated patients were 82% vs. 70% (p = 0.17). bi CEBPA- mutated patients showed a longer relapse free survival (RFS) (median RFS 9.4 years vs. 1.5 years, p = 0.021) and a lower cumulative incidence of relapse (CIR) compared to mo CEBPA- mutated patients. These differences in OS and RFS were confirmed after adjustment for known clinical and molecular prognostic factors. In this long-term observation we confirmed the favorable prognostic outcome of patients with bi CEBPA mutations compared to mo CEBPA- mutated CN-AML. The high probability of OS (81%) in younger patients is helpful to guide intensity of postremission therapy.