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新的食品和药物管理局批准和新兴的急性髓细胞白血病治疗选择。

New Food and Drug Administration-Approved and Emerging Novel Treatment Options for Acute Myeloid Leukemia.

机构信息

Rush University Medical Center, Chicago, Illinois.

Midwestern University Chicago College of Pharmacy, Downers Grove, Illinois.

出版信息

Pharmacotherapy. 2018 Nov;38(11):1143-1154. doi: 10.1002/phar.2180. Epub 2018 Oct 17.

DOI:10.1002/phar.2180
PMID:30220082
Abstract

Advancements in the treatment of acute myeloid leukemia (AML) have been sparse during the past several decades, and the disease continues to have a poor prognosis. However, in 2017 alone, four new medications approved by the U.S. Food and Drug Administration (FDA) for the treatment of AML reached the market. Midostaurin, liposomal cytarabine and daunorubicin, enasidenib, and gemtuzumab ogozamicin all showed benefit in respective clinical trials to gain approval for the treatment of AML in various patient populations. Additionally, many phase II and III clinical trials are currently ongoing to assess the safety and efficacy of other potential therapies for the treatment of AML. In this review, we summarize the results of the landmark clinical trials associated with the newly approved agents as well as the current ongoing clinical trials for the treatment of AML. A literature search was performed to retrieve data on agents currently being studied for use. Although the overall prognosis for patients with AML remains poor, the addition of the newly FDA-approved medications is a step in the right direction for a disease state that has proved difficult to treat.

摘要

在过去的几十年中,急性髓细胞白血病 (AML) 的治疗进展甚微,该疾病的预后仍然很差。然而,仅在 2017 年,就有四种新的药物获得美国食品和药物管理局 (FDA) 批准用于治疗 AML,进入市场。米哚妥林、脂质体阿糖胞苷和柔红霉素、依尼妥珠单抗、吉妥珠单抗奥唑米星均在各自的临床试验中显示出获益,从而获得批准,用于治疗各种患者人群的 AML。此外,目前正在进行许多 II 期和 III 期临床试验,以评估其他潜在治疗 AML 的疗法的安全性和疗效。在这篇综述中,我们总结了与新批准的药物相关的具有里程碑意义的临床试验结果,以及目前正在进行的 AML 治疗临床试验。进行了文献检索以检索正在研究用于治疗 AML 的药物的数据。尽管 AML 患者的总体预后仍然很差,但新的 FDA 批准药物的加入是朝着一种治疗效果证明困难的疾病状态迈出的正确方向。

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