摘要

The FDA approved ivosidenib (Tibsovo; Agios), a small-molecule inhibitor of isocitrate dehydrogenase (IDH)1 on July 20, 2018, for treatment of adults with relapsed or refractory acute myeloid leukemia (R/R AML) with susceptible IDH1 mutation as detected by an FDA-approved test. The efficacy of ivosidenib was established on the basis of complete remission (CR) + CR with partial hematologic recovery (CRh) rate, duration of CR + CRh, and conversion from transfusion dependence (TD) to transfusion independence (TI) in Study AG120-C-001, a single-arm trial. With median follow-up of 8.3 months for 174 adults with IDH1-mutated R/R AML treated with 500 mg ivosidenib daily, the CR + CRh rate was 33% [95% confidence interval (CI), 26-40], median duration of response was 8.2 (95% CI, 5.6-12) months, and conversion from TD to TI occurred in 37% of patients. These endpoints reflect short-term benefit in patients with an unmet medical need; long-term efficacy outcomes were not assessed. Serious adverse reactions (AR) in ≥5% of patients were differentiation syndrome (10%), leukocytosis (10%), and QT interval prolongation (7%). Common (≥20%) ARs of any grade were fatigue, leukocytosis, arthralgia, diarrhea, dyspnea, edema, nausea, mucositis, QT interval prolongation, rash, pyrexia, cough, and constipation. Assessment of long-term safety of ivosidenib is a condition of this approval.

译文

FDA 于 2018年7月20日批准了异柠檬酸脱氢酶 (IDH) 1 的小分子抑制剂 ivosidenib (Tibsovo; Agios), 用于治疗成人复发或难治性急性髓系白血病 (R/R AML),通过 FDA 批准的试验检测到易感 IDH1 突变。Ivosidenib 的疗效建立在完全缓解 (CR) CR 与部分血液系统恢复 (CRh) 率、 CR CRh 持续时间和输血依赖转化 (TD) 的基础上研究 AG120-C-001 中的输血独立性 (TI),一项单臂试验。对 8.3 名接受每日 174 mg ivosidenib 治疗的 IDH1-mutated R/R AML 患者进行的中位随访时间为 500 个月,CR CRh 率为 33% [95% 置信区间 (CI), 26-40],中位反应持续时间为 8.2 (95% CI,5.6-12) 个月,37% 的患者发生了从 TD 到 TI 的转换。这些终点反映了未满足医疗需求的患者的短期益处; 未评估长期疗效结果。≥ 5% 的患者出现严重不良反应 (AR) 为分化综合征 (10%) 、白细胞增多 (10%) 和 QT 间期延长 (7%)。任何级别的常见 (≥ 20%) ARs 是疲劳、白细胞增多、关节痛、腹泻、呼吸困难、水肿、恶心、粘膜炎、 QT 间期延长、皮疹、发热、咳嗽和便秘。对 ivosidenib 长期安全性的评估是批准的一个条件。

Ivosidenib

肿瘤 白血病 药物
概述  :  

在一些患有AML的人中,白血病细胞在IDH1或IDH2基因中具有突变。这些基因有助于细胞产生某些蛋白质,也称为IDH1和IDH2。其中一个基因的突变可以阻止血细胞以通常的方式成熟。被称为IDH抑制剂的靶向药物可以阻断这些IDH蛋白。这些药物似乎通过帮助白血病细胞成熟(分化)成更正常的细胞起作用。这些药物可用于治疗IDH1或IDH2突变的AML。 艾伏尼布是Agios Pharmaceuticals, Inc.研发的是一种IDH1抑制剂。它可用于治疗伴有I

Ivosidenib

释    义   n. 艾伏尼布

例    句   Between March 14, 2014 and May 12, 2017, 73 patients with mIDH1-cholangiocarcinoma were enrolled and received ivosidenib.2014年3月14日至2017年5月12日,选取mIDH1-胆管癌患者73例进行艾伏尼布治疗。

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