Background: Acute promyelocytic leukemia (APL) is a distinct subtype of acute myeloid leukemia (AML) characterized by a specific genetic translocation and favorable response to all-trans-retinoic acid (ATRA). This study aimed to determine the frequency of APL among childhood AML cases and evaluate treatment outcomes using ATRA combined with chemotherapy.
Methods: A retrospective study was conducted at the Central Teaching Hospital for Children (2001–
2006). Ninety-four children (≤15 years) diagnosed with AML were included. Among them, 22 were
identified with APL (FAB M3 subtype). Eight patients received ATRA plus chemotherapy, while 14
received chemotherapy alone. Data on clinical features, lab findings, treatment response,
complications, and survival were collected from medical records.
Result: APL represented 23.4% of AML cases. Median age was 7.5 years, with 54.5% female. Most patients were anemic (Hb < 8 g/dL in 80.2%) and thrombocytopenic (platelets < 20×10⁹/L in 63.6%). Among those treated with ATRA and chemotherapy, 87.5% achieved remission and 12.5% died during induction. In contrast, only 7.1% of patients treated with chemotherapy alone achieved remission, with 92.8% mortality during induction. Relapse occurred in 37.5% of the ATRA group. Common ATRA-related complications included mucocutaneous dryness (87%) and retinoic acid syndrome (25%).
Conclusions: APL is a common AML subtype in children. Combined ATRA and chemotherapy
significantly improves survival compared to chemotherapy alone. Expanding access to ATRA and molecular diagnostics is essential to improve outcomes in pediatric APL.