Department of Pharmacy, The Affiliated Hospital of Southwest Medical University, Luzhou, China.
School of Pharmacy, Southwest Medical University, Luzhou, China.
Clin Epigenetics. 2023 Jul 11;15(1):113. doi: 10.1186/s13148-023-01529-2.
To systematically evaluate the efficacy and safety of FDA-approved isocitrate dehydrogenase (IDH) inhibitors in the treatment of IDH-mutated acute myeloid leukemia (AML).
We used R software to conduct a meta-analysis of prospective clinical trials of IDH inhibitors in the treatment of IDH-mutated AML published in PubMed, Embase, Clinical Trials, Cochrane Library and Web of Science from inception to November 15th, 2022.
A total of 1109 IDH-mutated AML patients from 10 articles (11 cohorts) were included in our meta-analysis. The CR rate, ORR rate, 2-year survival (OS) rate and 2-year event-free survival (EFS) rate of newly diagnosed IDH-mutated AML (715 patients) were 47%, 65%, 45% and 29%, respectively. The CR rate, ORR rate, 2-year OS rate, median OS and median EFS of relapsed or refractory (R/R) IDH-mutated AML (394 patients) were 21%, 40%, 15%, 8.21 months and 4.73 months, respectively. Gastrointestinal adverse events were the most frequently occurring all-grade adverse events and hematologic adverse events were the most frequently occurring ≥ grade 3 adverse events.
IDH inhibitor is a promising treatment for R/R AML patients with IDH mutations. For patients with newly diagnosed IDH-mutated AML, IDH inhibitors may not be optimal therapeutic agents due to low CR rates. The safety of IDH inhibitors is controllable, but physicians should always pay attention to and manage the differentiation syndrome adverse events caused by IDH inhibitors. The above conclusions need more large samples and high-quality RCTs in the future to verify.
系统评价美国食品药品监督管理局（FDA）批准的异柠檬酸脱氢酶（IDH）抑制剂在治疗 IDH 突变急性髓系白血病（AML）中的疗效和安全性。
我们使用 R 软件对 2022 年 11 月 15 日之前在 PubMed、Embase、ClinicalTrials、Cochrane 图书馆和 Web of Science 上发表的关于 IDH 抑制剂治疗 IDH 突变 AML 的前瞻性临床试验进行了荟萃分析。
共有 10 篇文章（11 个队列）的 1109 例 IDH 突变 AML 患者纳入本荟萃分析。新诊断 IDH 突变 AML（715 例）的完全缓解（CR）率、客观缓解率（ORR）率、2 年总生存率（OS）率和 2 年无事件生存率（EFS）率分别为 47%、65%、45%和 29%。复发或难治性（R/R）IDH 突变 AML（394 例）的 CR 率、ORR 率、2 年 OS 率、中位 OS 和中位 EFS 分别为 21%、40%、15%、8.21 个月和 4.73 个月。胃肠道不良事件是最常见的所有级别不良事件，血液学不良事件是最常见的≥3 级不良事件。
IDH 抑制剂是 IDH 突变 R/R AML 患者有前途的治疗药物。对于新诊断的 IDH 突变 AML 患者，由于 CR 率低，IDH 抑制剂可能不是最佳治疗药物。IDH 抑制剂的安全性是可控的，但医生应始终注意并管理 IDH 抑制剂引起的分化综合征不良事件。上述结论需要未来更多的大样本和高质量 RCT 来验证。
