In vitro drug sensitivity and clinical efficacy of the HAG regimen in pediatric AML.

[BACKGROUND] Standard induction chemotherapy for acute myeloid leukemia (AML) using cytarabine combined with anthracyclines (epirubicin/IA or daunorubicin/DA) is effective but associated with severe adverse effects. The HAG regimen (low-dose homoharringtonine, cytarabine, G-CSF), known for lower toxicity, shows promise in adult AML, but its application in pediatric AML remains underexplored.

[METHODS] This study investigated the feasibility and efficacy of the HAG regimen in pediatric AML. Twenty-four newly diagnosed pediatric AML patients (excluding M3 subtype) were enrolled between August 2021 and November 2022. Patients received the HAG regimen as induction therapy. Drug sensitivity testing was performed to evaluate in vitro efficacy.

[RESULTS] After the first induction cycle with HAG, the overall complete remission (CR) rate was 75% (18/24). Patients classified as ELN favorable and intermediate-risk achieved a 100% CR rate. The 1-year overall survival (OS) rate was 83.3% ± 7.6%, and the 3-year OS rate was 75.0% ± 8.8%. The 1-year event-free survival (EFS) rate was 75.0% ± 8.8%, and the 3-year EFS rate was 66.1% ± 9.8%.

[CONCLUSION] The HAG regimen demonstrates high efficacy and holds great promise for pediatric AML. It achieves survival outcomes comparable to more intensive regimens while offering the significant advantage of reduced toxicity.