61P - Unveiling the distinctive molecular, clinical, and prognostic features of infant acute myeloid leukemia: An analysis study of pediatric AML datasets from the Children's Oncology Group

Background

Infant acute myeloid leukemia (AML), particularly in infants under 2 years old, presents unique clinical and biological characteristics, emphasizing the need for age-specific risk assessment and therapeutic approaches.

Methods

A retrospective study was conducted to investigate the clinical and biological features of children diagnosed with AML using data from four pediatric clinical trials by The Children's Oncology Group (COG). By comparing cytogenetic and molecular markers as well as treatment outcomes across different age groups, the study aimed to identify age-specific prognostic markers for infant AML, considering the potential for refining the current risk-stratification of infant AML.

Results

We identified infant AML patients as a clinical, molecular and prognosis distinct subgroup. The gene fusions (KMT2A::MLLT1 and KAT6A::r) independently and specifically predicted outcomes in infant AML and, when combined with BM (Bone marrow leukemic blast), PB (Peripheral blasts), MRD (Measurable residual disease), contributed to a superior three-tier risk model surpassing AML stratification systems currently employed in clinical trials, as well as the lncRNA expression and leukemic stem cell (LSC)-based models.

Conclusions

In conclusion, infant AML exhibits heterogeneity at clinical, molecular, and prognostic levels. The newly proposed system, which integrates simple clinical and gene-fusion features, holds promise as a valuable tool in informing clinical decision-making and guiding treatment strategies in routine clinical practice.

Editorial acknowledgement

Clinical trial identification

Legal entity responsible for the study

The author.

Funding

National Natural Science Foundation of China (Grant No. 81911530169).

Disclosure

The author has declared no conflicts of interest.