Sung Lillian, Buckstein Rena, Doyle John J, Crump Michael, Detsky Allan S
Department of Pediatrics, University of Toronto, Toronto, Ontario, Canada.
Cancer. 2003 Feb 1;97(3):592-600. doi: 10.1002/cncr.11098.
The role of allogeneic bone marrow transplantation (BMT) in the consolidation of young adults with acute myeloid leukemia (AML) with matched sibling donors (MSD) is controversial. Although BMT is associated with increased event free survival compared with intensive chemotherapy (CT) consolidation, BMT also is associated with increased treatment-related mortality and likely decreased quality of life and life expectancy in patients who do not develop recurrent disease.
The authors used decision analysis to compare three strategies for maximizing quality-adjusted life years (QALYs) in patients with AML in first remission with an MSD: BMT All, BMT None (consolidation CT only), or BMT in high-risk patients, as defined by baseline cytogenetic testing (Test strategy). A second decision-analysis tree was then constructed that compared BMT with CT specifically for patients with intermediate cytogenetics.
Using expected QALYs as the outcome measure, the Test, BMT All, and BMT None strategies were associated with 20.10 QALYs, 19.63 QALYs, and 18.38 QALYs, respectively. Thus, the Test strategy, with CT for low-risk patients and BMT for intermediate risk and high-risk patients, was expected to be the optimal strategy. In the intermediate cytogenetic decision analysis, although the expected QALY for BMT recipients was higher compared with CT recipients (19.78 QALYs vs. 18.75 QALYs), because of uncertainty in variable estimates, the optimal choice was less clear.
CT consolidation is a reasonable option for patients with AML who have favorable cytogenetics, even if an MSD is available. This model provides a framework from which patients with AML and their physicians can make decisions about consolidation therapy.
对于年轻的急性髓系白血病(AML)成年患者,在有匹配同胞供者(MSD)的情况下,异基因骨髓移植(BMT)在巩固治疗中的作用存在争议。尽管与强化化疗(CT)巩固治疗相比,BMT与无事件生存率的提高相关,但BMT也与治疗相关死亡率的增加有关,并且对于未复发的患者,其生活质量和预期寿命可能会降低。
作者采用决策分析方法,比较了三种使首次缓解且有MSD的AML患者的质量调整生命年(QALY)最大化的策略:全进行BMT、不进行BMT(仅采用巩固性CT)或根据基线细胞遗传学检测定义的高危患者进行BMT(检测策略)。然后构建了第二个决策分析树,专门比较了中等细胞遗传学特征患者的BMT与CT。
以预期QALY作为结果指标,检测策略、全进行BMT策略和不进行BMT策略分别与20.10个QALY、19.63个QALY和18.38个QALY相关。因此,对于低危患者采用CT、中危和高危患者采用BMT的检测策略预计是最佳策略。在中等细胞遗传学决策分析中,尽管BMT受者的预期QALY高于CT受者(19.78个QALY对18.75个QALY),但由于变量估计存在不确定性,最佳选择不太明确。
对于细胞遗传学特征良好的AML患者,即使有MSD,CT巩固治疗也是一个合理的选择。该模型为AML患者及其医生在巩固治疗决策方面提供了一个框架。