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比较减低毒性异基因造血细胞移植与常规化疗(免疫)治疗在复发或难治性 CLL 患者中的疗效:一项马尔可夫决策分析。

Comparing efficacy of reduced-toxicity allogeneic hematopoietic cell transplantation with conventional chemo-(immuno) therapy in patients with relapsed or refractory CLL: a Markov decision analysis.

机构信息

Department of Internal Medicine, Division of Hematology-Oncology and Bone Marrow Transplantation, American University of Beirut, Beirut, Lebanon.

出版信息

Bone Marrow Transplant. 2012 Sep;47(9):1164-70. doi: 10.1038/bmt.2012.71. Epub 2012 May 7.

DOI:10.1038/bmt.2012.71
PMID:22562081
Abstract

Despite therapeutic advances, relapsed/refractory CLL, particularly after fludarabine-based regimens, remains a major challenge for which optimal therapy is undefined. No randomized comparative data exist to suggest the superiority of reduced-toxicity allogeneic hematopoietic cell transplantation (RT-allo-HCT) over conventional chemo-(immuno) therapy (CCIT). By using estimates from a systematic review and by meta-analysis of available published evidence, we constructed a Markov decision model to examine these competing modalities. Cohort analysis demonstrated superior outcome for RT-allo-HCT, with a 10-month overall life expectancy (and 6-month quality-adjusted life expectancy (QALE)) advantage over CCIT. Although the model was sensitive to changes in base-case assumptions and transition probabilities, RT-allo-HCT provided superior overall life expectancy through a range of values supported by the meta-analysis. QALE was superior for RT-allo-HCT compared with CCIT. This conclusion was sensitive to change in the anticipated state utility associated with the post-allogeneic HCT state; however, RT-allo-HCT remained the optimal strategy for values supported by existing literature. This analysis provides a quantitative comparison of outcomes between RT-allo-HCT and CCIT for relapsed/refractory CLL in the absence of randomized comparative trials. Confirmation of these findings requires a prospective randomized trial, which compares the most effective RT-allo-HCT and CCIT regimens for relapsed/refractory CLL.

摘要

尽管在治疗上取得了进展,但复发/难治性 CLL,特别是在基于氟达拉滨的方案之后,仍然是一个主要的挑战,目前还没有定义最佳的治疗方法。没有随机对照数据表明降低毒性的异基因造血细胞移植(RT-allo-HCT)优于常规化疗(免疫)治疗(CCIT)。通过使用系统评价的估计值和对现有已发表证据的荟萃分析,我们构建了一个马尔可夫决策模型来研究这些竞争的治疗方法。队列分析表明,RT-allo-HCT 的结果更优,与 CCIT 相比,总生存期(以及 6 个月的质量调整生命期(QALE))有 10 个月的优势。尽管该模型对基本情况假设和转移概率的变化敏感,但 RT-allo-HCT 通过荟萃分析支持的一系列值提供了更优的总生存期。与 CCIT 相比,RT-allo-HCT 的 QALE 更高。这一结论对与异基因造血干细胞移植后状态相关的预期状态效用的变化敏感;然而,RT-allo-HCT 仍然是现有文献支持的价值的最佳策略。在没有随机对照试验的情况下,本分析提供了 RT-allo-HCT 和 CCIT 治疗复发/难治性 CLL 之间的结果的定量比较。需要一项前瞻性随机试验来证实这些发现,该试验比较了复发/难治性 CLL 最有效的 RT-allo-HCT 和 CCIT 方案。

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