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Research Article: Clinical outcomes and prognostic factors in pediatric acute myeloid leukemia with monocytic differentiation: a single-center cohort study from Egypt

Date Published: 2026-10-12

Abstract:
Acute myeloid leukemia (AML) with monocytic differentiation (FAB M4/M5 subtypes) is a distinct subtype of pediatric AML characterized by aggressive clinical behavior and common extramedullary involvement. Despite therapeutic advances, outcomes remain suboptimal, particularly in resource-limited settings. The study aimed to evaluate the clinical characteristics, treatment outcomes, and prognostic markers in pediatric patients with AML, FAB M4/M5 subtypes. A retrospective cohort study analyzing 193 patients with AML, FAB M4/M5 subtypes diagnosed at the Children’s Cancer Hospital, Egypt-57357 (CCHE-57357) between January 2016 and December 2021. The cohort comprised 131 patients with M4 and 62 with M5 morphology. Median follow-up was 62.0 months among survivors and 37.6 months for the entire cohort. Three- and five-year OS were 53.1% and 48.3%; three- and five-year EFS were 48.2% and 45.5%. Five-year CIR was 23.8%. Forty-eight patients (24.9%) died during induction. Multivariate Cox regression identified MRD positivity after first induction (HR 2.868, 95% CI 1.133–7.260, p = 0.026) and monosomy 7 (HR 6.206, 95% CI 2.692–14.306, p < 0.001) as independent adverse prognostic factors. Conversely, inv(16)(p13.1;q22) or t(16;16)(p13.1;q22); CBFB::MYH11 was an independent favorable prognostic factor (HR 0.253, 95% CI 0.108–0.592, p = 0.002). 14 high-risk pediatric AML patients underwent HSCT post-induction; three experienced adverse outcomes, comprising relapses ( n = 2), and acute GVHD ( n = 1). Genetic findings and treatment response were associated with outcome, supporting a complete diagnostic assessment. Improving survival requires precise risk stratification, MRD-guided therapy, and robust supportive care.

Introduction:
Acute myeloid leukemia (AML) with monocytic differentiation (FAB M4/M5 subtypes) is a distinct subtype of pediatric AML characterized by aggressive clinical behavior and common extramedullary involvement. Despite therapeutic advances, outcomes remain suboptimal, particularly in resource-limited settings. The study aimed to evaluate the clinical characteristics, treatment outcomes, and prognostic markers in pediatric patients with AML, FAB M4/M5 subtypes.

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