PETHEMA study links MRD-negative remission to survival in intensively treated AML

A multinational PETHEMA real-world study published in Leukemia assessed measurable residual disease (MRD) as a potential early endpoint of treatment efficacy in acute myeloid leukemia (AML). The analysis included 2,623 newly diagnosed patients fit for intensive therapy who achieved complete remission after one cycle. Investigators evaluated flow-cytometry MRD thresholds, associations with overall survival (OS), and whether differences in MRD-negative remission rates across induction regimens corresponded to differences in OS.

MRD was detected in 68% of patients despite morphological complete remission. Levels of at least 0.01% but below 0.1% were associated with inferior OS. MRD-negative remission defined using a 0.01% cutoff had independent prognostic value and correlated with OS at the individual-patient level (global odds ratio 1.23; 95% CI, 1.02–1.40). However, statistical significance was lost in specific subgroups defined by genetic risk and post-consolidation treatment. A relationship between treatment effects on MRD-negative remission rates and OS across induction regimens was generally observed.

These findings support further formal evaluation of MRD as an early efficacy endpoint in selected AML subgroups, rather than establishing it as a universally validated surrogate for survival. The observational design and restriction to patients achieving remission after one intensive cycle limit broader interpretation. Absolute survival estimates and detailed subgroup results were not provided in the available source text.