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首次缓解期成人急性髓系白血病的自体外周血干细胞移植——基于MRC AML10队列的意向性治疗分析及使用预测模型对结局的比较

Autologous peripheral blood stem cell transplantation in first remission adult acute myeloid leukaemia--an intention to treat analysis and comparison of outcome using a predictive model based on the MRC AML10 cohort.

作者信息

Ewing J C, Robertson J D, Kell W J, Burnett A K, Ryder D, Chang J, Morgenstern G R, Scarffe J H, Chopra R

机构信息

Department of Haematological Oncology, Christie Hospital, Manchester M20 4BX, UK.

出版信息

Hematology. 2003 Apr;8(2):83-90. doi: 10.1080/1024533031000090793.

DOI:10.1080/1024533031000090793
PMID:12745657
Abstract

The role of autologous peripheral blood stem cell transplantation (APBSCT) in acute myeloid leukaemia (AML) remains controversial. The current study evaluated the application of APBSCT in a large consecutive series of patients with untreated AML, and compared outcome with a predictive model based on MRC AML10 data. Of 148 evaluable patients, 118 patients entered complete remission (CR) after induction therapy comprising three cycles of daunorubicin, cytosine arabinoside and oral 6-thioguanine. Of these patients, 68 (57%) proceeded to consolidation therapy with two courses of intermediate dose cytosine arabinoside, and stem cell mobilisation, and 40 of these patients (34%) underwent the APBSCT procedure after high dose busulphan conditioning. Harvest quality was the main factor precluding APBSCT. Five-year event-free survival (EFS) in patients who achieved CR was 38% and in APBSCT patients was 57%. There were no transplant-related deaths. No significant differences were demonstrated between observed and expected outcomes at 1 and 2 years, based on the predictive model derived from the MRC AML10 study. These data therefore indicate that only a third of eligible adult patients will undergo APBSCT. However, the results demonstrate favourable survival in such patients, with no transplant-related mortality.

摘要

自体外周血干细胞移植(APBSCT)在急性髓系白血病(AML)中的作用仍存在争议。本研究评估了APBSCT在一大组未经治疗的连续AML患者中的应用,并将结果与基于MRC AML10数据的预测模型进行了比较。在148例可评估患者中,118例患者在接受包含柔红霉素、阿糖胞苷和口服6-硫鸟嘌呤三个周期的诱导治疗后进入完全缓解(CR)。在这些患者中,68例(57%)接受了两个疗程的中剂量阿糖胞苷巩固治疗、干细胞动员,其中40例患者(34%)在接受大剂量白消安预处理后进行了APBSCT手术。采集质量是排除APBSCT的主要因素。达到CR的患者5年无事件生存率(EFS)为38%,接受APBSCT的患者为57%。无移植相关死亡。根据MRC AML10研究得出的预测模型,在1年和2年时观察到的结果与预期结果之间没有显著差异。因此,这些数据表明,只有三分之一的符合条件的成年患者将接受APBSCT。然而,结果显示此类患者生存率良好,无移植相关死亡率。

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