The Floating Hospital for Children at Tufts Medical Center, Boston, Massachusetts; Department of Pediatrics, Tufts University School of Medicine, Boston, Massachusetts; Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, Massachusetts.
Pediatr Blood Cancer. 2014 Feb;61(2):269-75. doi: 10.1002/pbc.24739. Epub 2013 Aug 19.
We sought to better define the role of hematopoietic cell transplantation (HCT) in first remission (CR1) for high-risk pediatric acute myeloid leukemia (AML).
Outcomes were compared among patients aged less than 21 years with cytogenetically defined poor-risk AML treated with chemotherapy, matched related (MRD), or unrelated donor (URD) transplantation in CR1. Poor-risk cytogenetics was defined as monosomy 7/del7q, monosomy 5/del 5q, abnormalities of 3q, t(6;9)(p23;q34), or complex karyotype. Included are patients treated on Children's Oncology Group trials or reported to the Center for International Blood and Marrow Transplant Research from 1989 to 2006.
Of the 233 patients, 123 received chemotherapy, 55 received MRD HCT, and 55 received URD HCT. The 5-year overall survival from the time of consolidation chemotherapy or transplant conditioning was similar: chemotherapy (43% ± 9%), MRD (46% ± 14%), or URD (50% ± 14%), P = 0.99. Similarly, multivariate analysis demonstrated no significant differences in survival [(reference group = chemotherapy); MRD HR 1.08, P = 0.76; URD HR 1.13, P = 0.67] despite lower relapse risk with URD HCT (HR = 0.43, P = 0.01).
Our findings do not provide support for the preferential use of HCT over chemotherapy alone for children with cytogenetically defined poor-risk AML in CR1.
我们试图更好地定义造血细胞移植（HCT）在高危儿科急性髓细胞白血病（AML）首次缓解（CR1）中的作用。
在接受化疗、匹配相关（MRD）或无关供体（URD）移植的年龄小于 21 岁的伴有细胞遗传学定义的不良风险 AML 的患者中，比较了在 CR1 中的结局。不良风险细胞遗传学定义为单体 7/缺失 7q、单体 5/缺失 5q、3q 异常、t（6;9）（p23;q34）或复杂核型。包括在儿童肿瘤学组试验中接受治疗或在 1989 年至 2006 年期间向国际血液和骨髓移植研究中心报告的患者。
在 233 名患者中，123 名接受化疗，55 名接受 MRD HCT，55 名接受 URD HCT。巩固化疗或移植预处理后 5 年总生存率相似：化疗（43%±9%）、MRD（46%±14%）或 URD（50%±14%），P=0.99。同样，多变量分析表明，在生存方面没有显著差异[（参考组=化疗）；MRD HR1.08，P=0.76；URD HR1.13，P=0.67]，尽管 URD HCT 降低了复发风险（HR=0.43，P=0.01）。
我们的研究结果不支持在 CR1 中对细胞遗传学定义的不良风险 AML 患儿优先使用 HCT 而非单独化疗。
