Laboratory for Excellence in Systems Biomedicine of Pediatric Oncology, Department of Pediatric Hematology and Oncology, Chongqing Key Laboratory of Pediatric Metabolism and Inflammatory Diseases, Ministry of Education Key Laboratory of Child Development and Disorders, National Clinical Research Center for Child Health and Disorders, Children's Hospital of Chongqing Medical University, Chongqing, China; Department of Basic Medicine, School of Medicine, Foshan University, Foshan, Guangdong Province, China.
Laboratory for Excellence in Systems Biomedicine of Pediatric Oncology, Department of Pediatric Hematology and Oncology, Chongqing Key Laboratory of Pediatric Metabolism and Inflammatory Diseases, Ministry of Education Key Laboratory of Child Development and Disorders, National Clinical Research Center for Child Health and Disorders, Children's Hospital of Chongqing Medical University, Chongqing, China.
Cell Rep Med. 2024 Oct 15;5(10):101762. doi: 10.1016/j.xcrm.2024.101762. Epub 2024 Oct 3.
The efficacy of stem cell transplantation (SCT) in pediatric acute myeloid leukemia (pAML) remains unsatisfactory due to the limitations of existing prognostic models in predicting efficacy and selecting suitable candidates. This study aims to develop a cytomolecular risk stratification-independent prognostic model for SCT in pAML patients at CR1 stage. The pAML SCT model, based on age, KMT2A rearrangement (KMT2A-r), and minimal residual disease at end of course 1 (MRD1), effectively classifies patients into low-, intermediate-, and high-risk groups. We validate the effectiveness in an internal validation cohort and in four external validation cohorts, consisting of different graft sources and donors. Moreover, by incorporating the FMS-like tyrosine kinase 3/internal tandem duplication (FLT3/ITD) allelic ratio, the pAML SCT model is refined, enhancing its ability to effectively select suitable candidates. We develop a simple and robust risk stratification model for pAML patients undergoing SCT, to aid in risk stratification and inform pretransplant decision-making at CR1 stage.
由于现有预后模型在预测疗效和选择合适的候选者方面存在局限性，干细胞移植 (SCT) 在儿科急性髓细胞白血病 (pAML) 中的疗效仍不尽人意。本研究旨在为 CR1 期 pAML 患者的 SCT 开发一种与细胞分子风险分层无关的预后模型。基于年龄、KMT2A 重排 (KMT2A-r) 和第 1 疗程末微小残留病 (MRD1) 的 pAML SCT 模型，可有效地将患者分为低危、中危和高危组。我们在内部验证队列和四个外部验证队列中验证了其有效性，这些队列包含不同的移植物来源和供者。此外，通过纳入 FMS 样酪氨酸激酶 3/内部串联重复 (FLT3/ITD) 等位基因比，pAML SCT 模型得到了改进，提高了其有效选择合适候选者的能力。我们为接受 SCT 的 pAML 患者开发了一种简单而稳健的风险分层模型，以帮助在 CR1 期进行风险分层，并为移植前决策提供信息。
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