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阿扎胞苷治疗急性髓系白血病患者:意大利同情用药项目 82 例患者的报告。

Azacitidine for the treatment of patients with acute myeloid leukemia: report of 82 patients enrolled in an Italian Compassionate Program.

机构信息

Institute of Hematology, Tor Vergata Foundation Polyclinic, Rome, Italy.

出版信息

Cancer. 2012 Feb 15;118(4):1014-22. doi: 10.1002/cncr.26354. Epub 2011 Jul 14.

DOI:10.1002/cncr.26354
PMID:21761399
Abstract

BACKGROUND

The efficacy of azacitidine for the treatment of high-risk myelodysplastic syndromes has prompted the issue of its potential role even in the treatment of acute myeloid leukemia (AML).

METHODS

The authors analyzed 82 patients with AML who were diagnosed according to World Health Organization criteria. The median patient age was 72 years (range, 29-87 years), and 27 patients (33%) had secondary AML. Of 62 patients with evaluable cytogenetics, 18 patients (29%) had a poor-risk karyotype, and 44 patients (71%) had an intermediate karyotype. Thirty-five patients (43%) received azacitidine as front-line treatment, and 47 patients (57%) had previously received 1 or more line of chemotherapy.

RESULTS

The overall response rate was 32% (26 of 82 patients) and included 12 (15%) complete remissions (CRs), 4 (5%) CRs with incomplete blood count recovery (CRi), and 10 (12%) partial responses (PRs). Responses were observed more frequently among untreated patients compared with pretreated patients; in fact, 17 of 35 untreated patients (48%) responded, including 11 responses (31%) classified as CR/CRi. Conversely, only 9 of 47 pretreated patients (19%) responded, including 5 responses (11%) that were classified as CR/Cri. The response rate was significantly higher for untreated patients (P = .006) and those who had white blood cell counts <10 × 10(9) /L (P = .006). For untreated patients who achieved a response, the median overall response duration was 13 months, and the 1-year and 2-years overall survival rates were 58% and 24%, respectively.

CONCLUSIONS

The current results indicated that azacitidine promises to be an effective therapy for elderly patients with untreated AML and with white blood cell counts <10 × 10(9) /L.

摘要

背景

阿扎胞苷治疗高危骨髓增生异常综合征的疗效促使人们关注其在急性髓系白血病(AML)治疗中的潜在作用。

方法

作者分析了 82 例按世界卫生组织标准诊断的 AML 患者。患者中位年龄为 72 岁(范围,29-87 岁),27 例(33%)为继发性 AML。在 62 例可评估细胞遗传学的患者中,18 例(29%)存在不良核型,44 例(71%)存在中危核型。35 例(43%)患者接受阿扎胞苷作为一线治疗,47 例(57%)患者既往接受过 1 线或多线化疗。

结果

总缓解率为 32%(82 例患者中有 26 例),包括 12 例(15%)完全缓解(CR)、4 例(5%)CR 伴不完全血细胞计数恢复(CRi)和 10 例(12%)部分缓解(PR)。未治疗患者的缓解率高于治疗患者;实际上,35 例未治疗患者中有 17 例(48%)缓解,包括 11 例(31%)被归类为 CR/CRi。相反,47 例治疗患者中仅有 9 例(19%)缓解,包括 5 例(11%)被归类为 CR/Cri。未治疗患者的缓解率显著高于治疗患者(P=.006)和白细胞计数<10×10(9)/L 的患者(P=.006)。对于获得缓解的未治疗患者,中位总缓解持续时间为 13 个月,1 年和 2 年总生存率分别为 58%和 24%。

结论

目前的结果表明,阿扎胞苷有望成为白细胞计数<10×10(9)/L 的未经治疗的老年 AML 患者的有效治疗方法。

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