ArticleFrontiers in oncology2026
Superior long-term survival with acceptable safety of ATG/G-CSF-based haplo-HSCT with intensified BU+MEL/TT conditioning in CR Pediatric Non-DS-AMKL.
Article in Frontiers in oncology, 2026. 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: Non-Down syndrome acute megakaryoblastic leukemia (non-DS-AMKL) is a rare but high-risk subtype of pediatric AML with dismal outcomes. Allo-HSCT in first complete remission (CR1) is recommended as the preferred strategy for improving survival, yet the benefit of BU/CY intensification with melphalan (MEL) or thiotepa (TT) remains unclear. Methods: We retrospectively analyzed 70 pediatric non-DS-AMKL patients undergoing ATG/G-CSF-based haplo-HSCT between March 2010 and February 2024, comparing standard BU/CY (n=25) with intensified BU+MEL/TT (n=45). Endpoints included OS, LFS, RI, NRM, and TRM. Survival was estimated by Kaplan-Meier and compared with the log-rank test, while cumulative incidences were analyzed using Gray's test. Cox proportional hazards and Fine-Gray competing-risk regression models were applied for univariate and multivariate analyses, as appropriate. Results: Baseline clinical characteristics were comparable between groups, whereas graft CD34+ (median, 10.40×10 Conclusions: In pediatric non-DS-AMKL, intensified conditioning (BU+MEL/TT) independently improved OS and LFS in patients transplanted in CR without increasing early NRM. Pre-transplant NR/PR status was the strongest adverse factor, markedly increasing RI and NRM, underscoring the importance of achieving remission before transplantation.
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