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急性早幼粒细胞白血病的早期死亡率和总生存率:真实世界数据与临床试验结果相符吗?

Early mortality and overall survival in acute promyelocytic leukemia: do real-world data match results of the clinical trials?

机构信息

Department of Internal Medicine, Division of Oncology and Hematology, University of Nebraska Medical Center, Omaha, NE, USA.

Fred and Pamela Buffett Cancer Center, University of Nebraska Medical Center, Omaha, NE, USA.

出版信息

Leuk Lymphoma. 2021 Aug;62(8):1949-1957. doi: 10.1080/10428194.2021.1894651. Epub 2021 Mar 12.

DOI:10.1080/10428194.2021.1894651
PMID:33711907
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9429085/
Abstract

Acute promyelocytic leukemia (APL) boasts overall survival (OS) of >90% at 3 years and early mortality of <5% in recent clinical trials. Using a large National Cancer Database, we performed analysis of 7190 adults with APL to determine whether one-month mortality and OS of patients with APL treated in real-world practices mirror outcomes noted in clinical trials. Only 64% of total patients received multi-agent therapy; 32% received either single-agent therapy or no therapy at all. One-month mortality was 6% for patients ≤18 years, 6% for 19-40 years, 10% for 41-60 years, and 21% for >60 years. OS at 1- and 3-year were 81% and 75%, respectively. In a multivariate analysis, age ≤ 40 years, treatment at academic center, use of multi-agent therapy, and diagnosis after 2009 conferred better OS. In this largest database study in APL till date, we demonstrated an overall improvement in OS over time but challenges still exist in translating successes of clinical trials to real-world practices.

摘要

急性早幼粒细胞白血病 (APL) 在最近的临床试验中 3 年的总生存率 (OS) 超过 90%,早期死亡率<5%。我们使用大型国家癌症数据库,对 7190 名 APL 成年患者进行了分析,以确定在真实世界实践中治疗的 APL 患者的一个月死亡率和 OS 是否反映了临床试验中的结果。只有 64%的总患者接受了多药物治疗;32%的患者接受了单一药物治疗或根本没有治疗。≤18 岁的患者一个月死亡率为 6%,19-40 岁的患者为 6%,41-60 岁的患者为 10%,>60 岁的患者为 21%。1 年和 3 年的 OS 分别为 81%和 75%。在多变量分析中,年龄≤40 岁、在学术中心治疗、使用多药物治疗以及 2009 年后诊断与更好的 OS 相关。在迄今为止 APL 最大的数据库研究中,我们证明了 OS 随着时间的推移总体上有所改善,但将临床试验的成功转化为真实世界的实践仍然存在挑战。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5cb3/9429085/3f57f853dc51/nihms-1829482-f0002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5cb3/9429085/4614ab65f50c/nihms-1829482-f0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5cb3/9429085/3f57f853dc51/nihms-1829482-f0002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5cb3/9429085/4614ab65f50c/nihms-1829482-f0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5cb3/9429085/3f57f853dc51/nihms-1829482-f0002.jpg

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