高危急性髓系白血病的造血干细胞移植结局：儿童肿瘤协作组报告
Hematopoietic Stem Cell Transplantation Outcomes for High-Risk AML: A Report From the Children's Oncology Group.
作者信息
Huang Benjamin J, Meyer Lauren K, Alonzo Todd A, Wang Yi-Cheng, Lamble Adam J, Ries Rhonda E, Wang Weijie, Hirsch Betsy, Raca Gordana, Ma Xiaotu, Gamis Alan S, Aplenc Richard, Kolb E Anders, Cooper Todd M, Tarlock Katherine, Loken Michael R, Meshinchi Soheil, Chewning Joseph H, Woods William G, Horan John T
机构信息
Department of Pediatrics, University of California San Francisco, San Francisco, CA.
Helen Diller Family Comprehensive Cancer Center, University of California San Francisco, San Francisco, CA.
出版信息
J Clin Oncol. 2025 Jun 10;43(17):1961-1971. doi: 10.1200/JCO-24-01841. Epub 2025 Apr 28.
PURPOSE
Hematopoietic stem cell transplantation (HSCT) is used as consolidation for pediatric patients with high-risk AML in first complete remission (CR1). The definition of high-risk AML has evolved considerably over the past two decades with the successive identification of new unfavorable risk factors. We conducted a cross-study analysis to determine whether HSCT improves the outcomes of patients with contemporarily defined high-risk AML.
METHODS
We combined data from AAML0531 and AAML1031, the last two phase III clinical trials completed by the Children's Oncology Group (COG). These two trials established the prognostic importance of measurable residual disease (MRD) and several high-risk cryptic cytogenetic/molecular (CM) alterations, which were applied to reclassify patients in the current COG phase III clinical trial, AAML1831. We compared the outcomes after HSCT in CR1 with those after chemotherapy alone in CR1 in the redefined high-risk group.
RESULTS
Our study cohort comprised 463 patients with high-risk CM alterations and 72 patients with standard-risk (SR) CM results with positive MRD at end of induction I. In all, 33.9% and 45.8% of these groups underwent HSCT in CR1, respectively. HSCT was associated with decreased relapse and improved disease-free survival (DFS) in both groups. In the high-risk CM group, 5-year DFS was 26.0% (95% CI, 20.6 to 31.6) and 49.8% (95% CI, 41.7 to 57.4; < .001) in patients receiving chemotherapy alone and HSCT, respectively. In the SR CM and MRD+ groups, DFS was 16.9% (95% CI, 4.3 to 36.7) compared with 50.9% (95% CI, 32.7 to 66.5; = .032). HSCT was also associated with improvement in outcomes based on multivariable analysis and across subgroups defined by clinical trial and by high-risk CM subtype, with the exception of chromosome 7 or 5 loss.
CONCLUSION
HSCT was associated with improved outcomes in pediatric patients with contemporarily defined high-risk AML.
目的
造血干细胞移植（HSCT）被用作初治完全缓解（CR1）的高危急性髓系白血病（AML）患儿的巩固治疗。在过去二十年中，随着新的不良风险因素相继被发现，高危AML的定义有了很大演变。我们进行了一项跨研究分析，以确定HSCT是否能改善当代定义的高危AML患者的预后。
方法
我们合并了儿童肿瘤协作组（COG）完成的最后两项III期临床试验AAML0531和AAML1031的数据。这两项试验确立了可测量残留病（MRD）以及几种高危隐匿性细胞遗传学/分子（CM）改变的预后重要性，这些因素被应用于重新分类当前COG III期临床试验AAML1831中的患者。我们比较了重新定义的高危组中CR1期接受HSCT后的预后与仅接受化疗后的预后。
结果
我们的研究队列包括463例有高危CM改变的患者和72例诱导I期结束时MRD呈阳性且CM结果为标准风险（SR）的患者。在这些组中，分别有33.9%和45.8%的患者在CR1期接受了HSCT。HSCT与两组患者复发率降低和无病生存期（DFS）改善相关。在高危CM组中，仅接受化疗和接受HSCT的患者5年DFS分别为26.0%（95%CI，20.6至31.6）和49.8%（95%CI，41.7至57.4；P<.001）。在SR CM和MRD+组中，DFS分别为16.9%（95%CI，4.3至36.7）和50.9%（95%CI，32.7至66.5；P =.032）。基于多变量分析以及在由临床试验和高危CM亚型定义的亚组中，HSCT也与预后改善相关，但7号或5号染色体缺失的情况除外。
结论
HSCT与当代定义的高危AML患儿预后改善相关。
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