Allogeneic hematopoietic stem cell transplantation could overcome the poor prognosis of DNMT3AmutNPM1mutFLT3-ITDmut in acute myeloid leukemia: real-world multicenter analysis in China

This paper explores the clinical outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for patients with triple-mutated acute myeloid leukemia (AML), a condition marked by mutations in DNMT3A, NPM1, and FLT3-ITD genes. Despite being classified as intermediate-risk by the European LeukemiaNet (ELN) 2022 risk classification, triple-mutated AML generally has a poor prognosis.

The study involved 53 patients with intermediate-risk triple-mutated AML who received allo-HSCT between January 2017 and June 2022. Patient data was collected from multiple centers in China, and the study was approved by the institutional review board of each participating hospital.

The research found that the 1.5-year cumulative incidence of relapse after allo-HSCT was 11.9%, and this rate was comparable between patients who were minimal residual disease (MRD) positive and MRD negative before transplantation. The 1.5-year probabilities of leukemia-free survival (LFS) and overall survival (OS) were 80.3% and 81.8%, respectively. Importantly, no significant risk factors for relapse were identified in multivariate analysis.

The study suggests that allo-HSCT may significantly improve clinical outcomes for patients with triple-mutated AML, reducing relapse rates and improving survival probabilities. The researchers noted that most patients (94.3%) achieved complete remission before allo-HSCT, with 71.7% being MRD negative.

However, the study's limitations include a small sample size and the retrospective nature of the analysis. Future prospective multicenter studies are recommended to confirm these findings and explore the potential benefits of novel therapies in this patient population.

The research highlights the potential of allo-HSCT as an effective treatment option for triple-mutated AML patients. The findings indicate that this procedure could overcome the poor prognosis typically associated with this condition. The study also underscores the importance of MRD status before transplantation, as most patients were MRD negative, which may have contributed to the favorable outcomes.

In conclusion, this real-world multicenter analysis in China provides valuable insights into the treatment of triple-mutated AML. The results suggest that allo-HSCT could be a promising approach for these patients, offering improved survival rates and lower relapse probabilities. Further research is needed to validate these findings and explore additional therapeutic strategies for this specific subset of AML patients.

DOI: 10.1007/s11684-024-1091-5