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评估 glasdegib 治疗急性髓细胞性白血病的效果。

An evaluation of glasdegib for the treatment of acute myelogenous leukemia.

机构信息

Hospices Civils de Lyon, Hematology Department, Lyon-Sud University Hospital, Pierre Bénite, France.

出版信息

Expert Opin Pharmacother. 2020 Apr;21(5):523-530. doi: 10.1080/14656566.2020.1713094. Epub 2020 Feb 6.

Abstract

: Despite recent advances in the treatment of adult acute myelogenous leukemia (AML), the overall outcome remains dismal especially in high-risk AML patients, including the elderly and the relapsed/refractory populations. In this setting, various clinical trials have recently explored novel therapeutic agents either used alone or in combination with intensive chemotherapy or low-intensity treatments.: The current paper reviews the clinical development of glasdegib, a selective inhibitor of the Hedgehog signaling pathway through binding to its target SMO, for the treatment of AML.: Glasdegib confirmed its efficacy and showed an acceptable tolerability, especially when used in combination either with '3 + 7' chemotherapy or with low-intensity therapies. In 2018, glasdegib was approved by the Food and Drug Administration (FDA) in combination with low-dose cytarabine for the treatment of newly diagnosed AML in patients older than 75 years or presenting with severe comorbidities.

摘要

尽管近年来在成人急性髓细胞性白血病(AML)的治疗方面取得了进展,但总体结果仍然不容乐观,尤其是在高危 AML 患者中,包括老年人和复发/难治性患者。在这种情况下,最近的各种临床试验已经探索了新型治疗药物,这些药物单独使用或与强化化疗或低强度治疗联合使用。本文综述了 Hedgehog 信号通路的选择性抑制剂 glasdegib 的临床开发情况,该抑制剂通过与靶标 SMO 结合来治疗 AML。Glasdegib 证实了其疗效,并表现出可接受的耐受性,尤其是与“3+7”化疗或低强度治疗联合使用时。2018 年,glasdegib 获得美国食品和药物管理局(FDA)批准,与低剂量阿糖胞苷联合用于治疗年龄大于 75 岁或伴有严重合并症的新诊断 AML 患者。

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