奥拉帕利单药治疗 BRCA 突变晚期卵巢癌的疗效和安全性
Olutasidenib: from bench to bedside.
机构信息
Division of Hematology, Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL.
出版信息
Blood Adv. 2023 Aug 22;7(16):4358-4365. doi: 10.1182/bloodadvances.2023009854.
Abstract
The discovery of isocitrate dehydrogenase 1 (IDH1) mutations in acute myeloid leukemia (AML) and the resounding success of molecularly targeted therapies in related myeloid malignancies swiftly prompted the development of IDH1mut inhibitors. Olutasidenib (formerly known as FT-2102) is an orally administered novel IDH1mut inhibitor that entered clinical development in 2016, proceeded briskly through the developmental process, and was granted regular approval to treat patients with R/R IDH1mut AML on 1 December 2022. Single agent olutasidenib, a potent and selective IDH1mut inhibitor, demonstrated highly durable remission rates along with meaningful outcomes, such as transfusion independence, in patients with R/R IDH1mut AML. This review will examine the preclinical and clinical development and the positioning of olutasidenib in the IDH1mut AML treatment landscape.
摘要
异柠檬酸脱氢酶 1（IDH1）突变在急性髓系白血病（AML）中的发现，以及相关髓系恶性肿瘤中分子靶向治疗的巨大成功，迅速促使 IDH1mut 抑制剂的开发。奥拉帕尼（前身为 FT-2102）是一种口服新型 IDH1mut 抑制剂，于 2016 年进入临床开发，在开发过程中进展迅速，并于 2022 年 12 月 1 日获得常规批准，用于治疗复发/难治性 IDH1mut AML 患者。单一药物奥拉帕尼是一种有效的、选择性的 IDH1mut 抑制剂，在复发/难治性 IDH1mut AML 患者中表现出高度持久的缓解率，并具有显著的疗效，如输血独立性。这篇综述将探讨奥拉帕尼的临床前和临床开发情况，以及其在 IDH1mut AML 治疗领域的定位。
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