Positive outcomes associated with stem cell transplantation in pediatric/AYA patients who receive FLAG-IDA-venetoclax for high-risk AML

作者：Irtiza Sheikh, Reeja Raj, Olayinka Okeleji, Samanta Catueno, Aaron E. Fan, David McCall, Amber Gibson, Miriam B. Garcia, Jeremy Connors, Michael Roth, César Núnêz, Demetrios Petropoulos, Branko Cuglievan, Priti Tewari · 发表于：Blood · 年份：2025 · DOI：10.1182/blood-2025-5156 · 研究领域：Acute Myeloid Leukemia Research、Acute Lymphoblastic Leukemia research、CAR-T cell therapy research

Abstract Background: BCL-2 inhibitors like venetoclax have demonstrated encouraging remission rates when combined with fludarabine, cytarabine, idarubicin, and G-CSF (FLAG-IDA), enabling more patients to proceed to stem cell transplantation (SCT). However, Venetoclax (Ven) use in frontline pediatric AML remains investigational, and its effects on post-transplant immune reconstitution, engraftment kinetics, and graft-versus-host disease (GVHD) risk are not well defined. Recent data from Raj et al. demonstrate that the combined upfront use of a regimen such as FLAG-IDA-Ven in pediatric and adolescents and young adults (AYAs) with AML is associated with rapid remission, allowing the regimen to serve as a bridge to stem cell transplantation (SCT). Here, we conducted a retrospective review of the same cohort to evaluate transplant-related outcomes in children and AYAs with high-risk AML who received FLAG-IDA-Ven prior to SCT, focusing on engraftment, GVHD incidence, and early post-transplant recovery. Methods: A retrospective chart review was performed at the University of Texas MD Anderson Cancer Center to evaluate adverse events (AEs), engraftment, GVHD rates, and infectious complications in pediatric and AYA patients who received FLAG-IDA-Ven prior to SCT. Response was assessed using the revised International Working Group Response Criteria for AML, and measurable residual disease (MRD) was measured by multiparameter flow cytometry (sensitivity: 0.01%). Results: Of the 12 pedia...