Proposed Modifications to Prognostic Classification of AML Patients Treated With Intensive Chemotherapy Based on Recent Real-World Data.
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METHODS: The outcomes of all newly diagnosed acute myeloid leukemia (AML) patients treated with intensive chemotherapy (IC) at a single center between 2018 and 2024 were evaluated. RESULTS: The complete remission (CR) rates were not significantly different between ELN2022 favorable (fav) versus intermediate (int) risk patients. Of patients achieving CR, 81% of int risk and 85% of adverse (adv) risk patients intended for allogeneic stem cell transplant (HSCT) underwent HSCT in CR1. The overall survival (OS) and relapse-free survival (RFS) were not significantly different between ELN fav versus int risk patients. In contrast, OS was significantly worse in ELN adv risk patients, including those who underwent HSCT, due to both lower CR and higher relapse rates. Within the ELN adv group, patients with RUNX1 mutations had a superior OS compared with other myelodysplasia-related (MR) mutations or adv risk cytogenetics without RUNX1. TP53 mutated patients had the worst outcome. Based on these results, a transplant-adjusted prognostic scoring system was devised, incorporating HSCT into an integrated postremission strategy. In this model, ELN fav and int risk patients were considered good risk, RUNX1mut and KMT2Ar patients intermediate risk, other ELN adv non-TP53 mutated patients poor risk, and TP53 mutated patients very poor risk. The 5-year OS in these groups was 78%, 54%, 22% and 0%, respectively (P < .001). CONCLUSIONS: This revised classification provides a better discrimination of prognostic groups as compared with ELN2022, likely due to the high rate of HSCT now achievable for ELN int risk patients. However, results in ELN adv risk patients remain suboptimal.