ArticleFrontiers in immunology2025
Azacitidine as maintenance therapy in pediatric
Article in Frontiers in immunology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Relapse remains a major challenge in pediatric acute myeloid leukemia (AML), particularly in patients ineligible for hematopoietic stem cell transplantation (HSCT). Hypomethylating agents like azacitidine are hypothesized to target residual disease, but their efficacy and safety as maintenance therapy in Methods: In this retrospective cohort study, 78 pediatric patients with Results: At a median follow-up of 34.6 months, azacitidine maintenance showed comparable EFS (77.7% vs. 77.0%, p = 0.688), OS (89.7% vs. 85.0%, p=0.368) and CIR (22.3% vs. 21.0%, p=0.838) to observation in the overall cohort. Subgroup analysis suggested a non-significant trend toward improved EFS (85.1% vs. 69.3%, p=0.198) and OS (92.9% vs. 81.6%, p=0.304) in intermediate-risk patients. However, among the 17 patients with core-binding factor AML (CBF-AML) and baseline fusion transcripts ≥0.1%, azacitidine maintenance (n=9) showed significantly superior EFS and CIR compared to observation (n=8) (EFS: 100% vs. 62.5%, p=0.048; CIR: 0.0% vs. 40.0%, p = 0.042). Although 40.7% of patients experienced grade 2-4 myelosuppression, all completed the treatment without dose reductions. Conclusion: Azacitidine maintenance therapy may sustain molecular remission in specific subgroups of pediatric AML, particularly CBF-AML patients with persistent MRD after induction who are ineligible for HSCT. However, the potential benefits must be weighed against the toxicity profile. The optimal dosing and scheduling require further investigation, and broader application warrants validation through larger, prospective, randomized controlled trials.
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