Decitabine priming + low-dose chemotherapy in pediatric R/R AML

Results from the prospective, multicenter, phase II study (ChiCTR1800015872), evaluating decitabine priming combined with low-dose chemotherapy (idarubicin + cytarabine + granulocyte colony-stimulating factor; DP‑IAG) in 101 pediatric patients aged <18 years with relapsed/refractory (R/R) acute myeloid leukemia (AML) were published in Haematologica by Fan et al. The primary endpoint was complete remission (CR) rate, including incomplete hematologic recovery (CRi), after DP‑IAG reinduction.

Key data: Following one course of DP‑IAG, 73.3% of patients achieved CR/CRi (95% confidence interval [CI], 64.6–81.9%). At a median follow-up of 36.4 months, the estimated 3-year overall survival (OS) was 60.8% (95% CI, 51.9–71.2%) and 3-year event-free survival (EFS) was 49.8% (95% CI, 40.9–60.7%). Patients who exhibited CR/CRi following reinduction therapy demonstrated greater 3-year OS and EFS vs patients with a partial response or no response (both p < 0.001). After one cycle of DP‑IAG, 20 patients proceeded to allogeneic hematopoietic stem cell transplantation (allo-HSCT); 41 patients received allo-HSCT after two or more cycles of therapy. The most frequent non-hematologic treatment-related adverse events were infections, including bacteremia and pneumonia. All patients experienced Grade 4 hematologic toxicity.

Key learning: DP-IAG demonstrated high remission rates with an acceptable safety profile in pediatric patients with R/R AML, supporting its potential role as a salvage regimen and feasible bridge to allo-HSCT.