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复发或难治性急性髓系白血病(rAML)患儿的治疗结果:儿童白血病治疗进展(TACL)联盟研究。

Outcome for children treated for relapsed or refractory acute myelogenous leukemia (rAML): a Therapeutic Advances in Childhood Leukemia (TACL) Consortium study.

机构信息

Therapeutic Advances in Childhood Leukemia Consortium, USC-CHLA Institute for Pediatric Clinical Research, Los Angeles, California, USA.

出版信息

Pediatr Blood Cancer. 2010 Sep;55(3):421-9. doi: 10.1002/pbc.22612.

Abstract

BACKGROUND

Current event-free survival (EFS) rates for children with newly diagnosed acute myeloid leukemia (AML) approach 50-60%. We hypothesize that further improvements in survival are unlikely to be achieved with traditional approaches such as dose intensive chemotherapy or hematopoietic stem cell transplants, since these therapies have been rigorously explored in clinical trials. This report highlights efforts to assess the response rates and survival outcomes after first or greater relapse in children with AML.

PROCEDURE

We performed a retrospective cohort review of pediatric patients with relapsed and refractory AML (rAML) previously treated at TACL institutions between the years of 1995 and 2004. Data regarding disease characteristics at diagnosis and relapse, treatment response, and survival was collected on 99 patients and 164 medullary relapses or treatment failures.

RESULTS

The complete response (CR) rate following the second therapeutic attempt was 56 +/- 5%. CR rates following a third treatment attempt was 25 +/- 8% while 17 +/- 7% achieved CR following the fourth through sixth treatments. The 5-year disease-free survival in patients achieving CR following a second therapeutic attempt was 43 +/- 7%. The 5-year EFS and overall survival (OS) rates for all patients receiving a second treatment attempt was 24 +/- 5% and 29 +/- 5%, respectively.

CONCLUSIONS

This CR rate following a second therapeutic attempt and OS rate in patients with rAML is consistent with the literature. There are limited published data of CR rates for subsequent relapses. Our data can serve as a historical benchmark to compare outcomes of future therapeutic trials in rAML against traditional chemotherapy regimens.

摘要

背景

新诊断为急性髓系白血病(AML)的儿童目前的无事件生存(EFS)率接近 50-60%。我们假设,通过传统方法(如强化剂量化疗或造血干细胞移植)进一步提高生存率的可能性不大,因为这些疗法已经在临床试验中得到了严格的探索。本报告重点介绍了评估 AML 儿童首次或更大复发后的缓解率和生存结果的努力。

程序

我们对 1995 年至 2004 年间在 TACL 机构接受治疗的复发和难治性 AML(rAML)儿科患者进行了回顾性队列研究。收集了 99 名患者和 164 例髓系复发或治疗失败患者的诊断和复发时疾病特征、治疗反应和生存数据。

结果

第二次治疗尝试后的完全缓解(CR)率为 56 +/- 5%。第三次治疗尝试后的 CR 率为 25 +/- 8%,而第四次至第六次治疗后的 CR 率为 17 +/- 7%。第二次治疗尝试后达到 CR 的患者 5 年无病生存率为 43 +/- 7%。接受第二次治疗尝试的所有患者的 5 年 EFS 和总生存率(OS)分别为 24 +/- 5%和 29 +/- 5%。

结论

rAML 患者第二次治疗尝试后的 CR 率和 OS 率与文献一致。随后复发的 CR 率的发表数据有限。我们的数据可以作为历史基准,用于比较未来 rAML 治疗试验与传统化疗方案的结果。

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