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低强度预处理异基因造血细胞移植治疗年轻急性髓系白血病患者:一项基于注册登记的研究

Reduced-intensity conditioning allogeneic hematopoietic cell transplantation for younger patients with acute myeloid leukemia: a registry-based study.

作者信息

Yanada M, Kurosawa S, Kobayashi T, Ozawa Y, Kanamori H, Kobayashi N, Sawa M, Nakamae H, Uchida N, Hashimoto H, Fukuda T, Hirokawa M, Atsuta Y, Yano S

机构信息

Fujita Health University School of Medicine, Toyoake, Japan.

National Cancer Center Hospital, Tokyo, Japan.

出版信息

Bone Marrow Transplant. 2017 Jun;52(6):818-824. doi: 10.1038/bmt.2016.358. Epub 2017 Jan 23.

Abstract

Clinical efficacy of allogeneic hematopoietic cell transplantation (HCT) using reduced-intensity conditioning (RIC) for younger patients remains unclear. We therefore performed a retrospective registry-based study to evaluate outcomes for patients with AML aged between 16 and 49 years who underwent RIC allogeneic HCT. Patients receiving RIC (N=125) showed significantly worse survival than those receiving myeloablative conditioning (MAC; N=1,554) (47.7% for RIC and 54.2% for MAC at 4 years, P=0.047). However, the difference became marginal after adjustment for patient characteristics (P=0.080), and inclusion in the multivariate analysis of the HCT comorbidity index or the propensity score for estimating the likelihood of choosing RIC or MAC further reduced statistical significance (P=0.371 and 0.206, respectively), indicating the existence of a selection bias against RIC. Nevertheless, outcomes for our patients receiving RIC were still acceptable, so that RIC constitutes a potential therapeutic option for younger AML patients who are deemed unsuitable for MAC. Subgroup analyses showed that patients aged between 40 and 49 years as well as those in first or second CR at the time of transplantation exhibited similar outcomes regardless of whether they were treated with RIC or MAC.

摘要

对于年轻患者,采用减低强度预处理(RIC)进行异基因造血细胞移植(HCT)的临床疗效仍不明确。因此,我们进行了一项基于登记处的回顾性研究,以评估年龄在16至49岁之间接受RIC异基因HCT的急性髓系白血病(AML)患者的预后。接受RIC的患者(N = 125)的生存率显著低于接受清髓性预处理(MAC;N = 1554)的患者(4年时RIC组为47.7%,MAC组为54.2%,P = 0.047)。然而,在对患者特征进行调整后,差异变得不显著(P = 0.080),并且将HCT合并症指数或估计选择RIC或MAC可能性的倾向评分纳入多变量分析后,统计学显著性进一步降低(分别为P = 0.371和0.206),表明存在对RIC的选择偏倚。尽管如此,我们接受RIC治疗的患者的预后仍然可以接受,因此RIC对于被认为不适合MAC的年轻AML患者构成了一种潜在的治疗选择。亚组分析显示,年龄在40至49岁之间以及移植时处于首次或第二次完全缓解(CR)的患者,无论接受RIC还是MAC治疗,其预后相似。

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