Is Acute Myeloblastic Leukemia in Children Under 2 Years of Age a Specific Entity? A Report from the FRENCH ELAM02
S Blais1,2, H Boutroux2,3, M Pasquet4
1Pediatric Department, Poissy Hospital, Poissy, France.
Insights
Infants with acute myeloid leukemia (AML) show distinct clinical and biological traits, including more frequent extra-medullary disease and KMT2A rearrangements. However, these differences did not impact overall survival or event-free survival in the ELAM02 protocol.
Area of Science:
- Pediatric Oncology
- Hematology
- Leukemia Research
Background:
- Infants (under 2 years) with acute myeloid leukemia (AML) exhibit unique clinical and biological characteristics compared to older children.
- Understanding these differences is crucial for identifying prognostic factors in infant AML.
Purpose of the Study:
- To describe the specific clinical and biological features of infants diagnosed with AML.
- To evaluate the impact of these features on the prognosis of infant AML patients.
Main Methods:
- Analysis of data from 438 newly-diagnosed AML patients in the ELAM02 protocol (March 2005-December 2011).
- Comparison of clinical and biological features, overall survival (OS), and event-free survival (EFS) between infants (n=103) and older children.
- Specific evaluation of extra-medullary presentation, skin lesions, central nervous system involvement, and KMT2A rearrangements.
Main Results:
- Infants showed a higher incidence of extra-medullary presentation, including skin lesions (15% vs 3%) and CNS involvement (26% vs 12%).
- KMT2A rearrangements were significantly more common in infants (55% vs 11%).
- Despite these differences, median 5-year OS (70.4% vs 71.4%) and 5-year EFS (67% vs 58%) were comparable between infants and older children.
Conclusions:
- Infants with AML constitute a distinct patient cohort with specific clinical and biological profiles.
- These unique features did not significantly affect survival outcomes within the ELAM02 protocol.
- Further research may be warranted to explore the implications of these distinct characteristics in infant AML management.
Abstract:
The clinical and biological characteristics of children under 2 years (infants) with acute myeloid leukemia (AML) are different from those of older children. We aimed to describe the specific characteristics of this population and the potential factors that influence the prognosis. We analyzed data concerning 438 children with newly-diagnosed AML treated in the ELAM02 protocol between March 2005 and December 2011, of which 103 were under 2 years old at diagnosis. The evaluation criteria were overall survival (OS) and event-free survival (EFS) of infants vs older children. The clinical and biological features were secondary criteria. Infants presented more frequent extra-medullary presentation than older children. They had a significantly higher proportion of skin lesions and central nervous system involvement (15% vs 3%, p < 0.0001 and 26% vs 12%, p = 0.0005, respectively). The global incidence of KMT2A rearrangements was nearly 55% for infants vs 11% for older children (p < 0.0001). Median 5-year OS was 70.4% for infants vs 71.4% for older children (p = 0.83). Five-year EFS was 67% for infants vs 58% for older children (p = 0.27). Infants with AML represent a cohort of patients with specific clinical and biological features. These remarkable differences had no significant impact on their outcome in the ELAM02 protocol.


