ABSTRACT

Introduction

Treatment of acute myeloid leukemia (AML) has changed dramatically in the past ten years with the approval of targeted agents, the first of which was the anti-CD33 antibody-drug conjugate gemtuzumab ozogamicin (GO). Despite withdrawal from the market after accelerated approval, GO was reapproved and now has a well-established role in treating select AML patients. CD33 has proven to be an important target for drug development in AML as evidenced by the improvement in survival with GO treatment.

Areas covered

The review summarizes the development of GO, its mechanism of action, initial studies and approval, withdrawal from the market, and subsequent reapproval after the results of several large randomized studies became available. We also provide an overview of its current role in the treatment landscape of AML.

Expert opinion

Multiple phase 3 trials with GO have established a significant benefit with GO in induction therapy for favorable risk AML. Additional studies support the use of GO in relapsed/refractory AML and APL. Despite the withdrawal of GO from the market after initial approval, GO has proven to improve survival of select AML patients when added to induction chemotherapy and in relapsed disease.

中文翻译：

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Gemtuzumab ozogamicin 用于治疗成人急性髓细胞白血病

摘要

介绍

在过去十年中，随着靶向药物的批准，急性髓性白血病 (AML) 的治疗发生了巨大变化，其中第一个是抗 CD33 抗体-药物偶联物吉妥珠单抗奥佐米星 (GO)。尽管在加速批准后退出了市场，但 GO 被重新批准，现在在治疗特定 AML 患者方面具有公认的作用。CD33 已被证明是 AML 药物开发的重要靶点，GO 治疗可提高生存率就证明了这一点。

涵盖的领域

该评价总结了 GO 的发展、其作用机制、初步研究和批准、退出市场以及随后在几项大型随机研究结果公布后的重新批准。我们还概述了其当前在 AML 治疗领域中的作用。

专家意见

GO 的多项 3 期试验已确定 GO 在有利风险 AML 的诱导治疗中具有显着益处。其他研究支持在复发/难治性 AML 和 APL 中使用 GO。尽管 GO 在初步批准后退出市场，但事实证明，当加入诱导化疗和复发性疾病时，GO 可以提高选定 AML 患者的生存率。