Background: As of 2018, no standard salvage regimen had been established for pediatric relapsed or refractory acute myeloid leukemia (R/R AML).Methods: In this prospective, multicenter Phase II trial, 101 evaluable patients under 18 years of age with R/R AML received one course of decitabine priming followed by low-dose idarubicin, cytarabine, and G-CSF (DP-IAG) as remission reinduction therapy. The primary objective was to determine the rate of complete remission, including remission with incomplete hematologic recovery (CR/CRi).Results: Seventy-four patients (73.3%; 95% CI: 64.6–81.9%) achieved CR/CRi and four patients achieved PR. The overall response rate (ORR) was 77.2% (95% CI: 68.9–85.5%). Among them, 20 proceeded to allogeneic hematopoietic stem cell transplantation (allo-HSCT) after reinduction I. Among the remaining patients, 55 underwent a second reinduction course with either DP-IAG (n = 33) or standard induction chemotherapy (n = 22). After this, 31 proceeded directly to allo-HSCT. At a median follow-up of 36.4 months (IQR: 10.3–51.3 months), the estimated 3-year overall survival rate was 60.8% (95% CI: 55.9–65.7%). Infections were the most common non-hematologic adverse events, and three patients died from treatment-related toxicity after the first DP-IAG course.Conclusions: These results support DP-IAG as an effective and tolerable salvage regimen, providing a feasible bridge to HSCT and a new reference point for future trials in pediatric R/R AML.

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2025
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