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通过靶向突变的异柠檬酸脱氢酶1（IDH1）改善急性髓系白血病（AML）在前期骨髓增殖性肿瘤（MPN）基础上的治疗结果。

Advancing the outcomes of AML out of antecedent MPN by targeting mutated IDH1.

作者信息

Pratz Keith W

机构信息

Hospital of University of Pennsylvania, Philadelphia, Pennsylvania, USA.

出版信息

Br J Haematol. 2025 Apr;206(4):1250-1252. doi: 10.1111/bjh.19959. Epub 2024 Dec 22.

DOI:10.1111/bjh.19959
PMID:39710967
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11985367/
Abstract

Outcomes of myeloproliferative neoplasms (MPN)-associated acute leukaemias are dismal with conventional therapy. Approximately 20% of MPN-associated acute leukaemias have mutations in isocitrate dehydrogenase (IDH). Olutasidenib, and inhibitor of IDH1, demonstrates important clinical benefits in MPN-associated leukaemia with IDH1 mutation. Commentary on: Botton et al. Olutasidenib demonstrates significant clinical activity in mutated IDH1 acute myeloid leukemia arising from a prior myeloproliferative neoplasm. Br J Haematol 2025; 206:1121-1128.

摘要

骨髓增殖性肿瘤（MPN）相关的急性白血病采用传统疗法的预后很差。约20%的MPN相关急性白血病存在异柠檬酸脱氢酶（IDH）突变。IDH1抑制剂奥芦他定尼在伴有IDH1突变的MPN相关白血病中显示出重要的临床益处。评论：博顿等人。奥芦他定尼在先前骨髓增殖性肿瘤引起的IDH1突变型急性髓系白血病中显示出显著的临床活性。《英国血液学杂志》2025年；206:1121 - 1128。

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本文引用的文献

1
Olutasidenib demonstrates significant clinical activity in mutated IDH1 acute myeloid leukaemia arising from a prior myeloproliferative neoplasm.奥拉帕尼在先前骨髓增殖性肿瘤引起的IDH1突变急性髓系白血病中显示出显著的临床活性。
Br J Haematol. 2025 Apr;206(4):1121-1128. doi: 10.1111/bjh.19944. Epub 2024 Dec 19.
2
Treatment approach and outcomes of patients with accelerated/blast-phase myeloproliferative neoplasms in the current era.当代加速/急变期骨髓增殖性肿瘤患者的治疗方法和结局。
Blood Adv. 2024 Jul 9;8(13):3468-3477. doi: 10.1182/bloodadvances.2024012880.
3
FDA Approval Summary: Ivosidenib in Combination with Azacitidine for Treatment of Patients with Newly Diagnosed Acute Myeloid Leukemia with an IDH1 Mutation.FDA 批准概要：ivosidenib 联合阿扎胞苷治疗新诊断的伴有 IDH1 突变的急性髓系白血病患者。
Clin Cancer Res. 2024 Apr 1;30(7):1226-1231. doi: 10.1158/1078-0432.CCR-23-2234.
4
A Phase Ib/II Study of Ivosidenib with Venetoclax ± Azacitidine in IDH1-Mutated Myeloid Malignancies.ivosidenib 联合 venetoclax ± 阿扎胞苷治疗 IDH1 突变型髓系恶性肿瘤的 Ib/II 期研究。
Blood Cancer Discov. 2023 Jul 5;4(4):276-293. doi: 10.1158/2643-3230.BCD-22-0205.
5
Response Rate, Event-Free Survival, and Overall Survival in Newly Diagnosed Acute Myeloid Leukemia: US Food and Drug Administration Trial-Level and Patient-Level Analyses.新发急性髓系白血病的缓解率、无事件生存和总生存：美国食品和药物管理局试验水平和患者水平分析。
J Clin Oncol. 2022 Mar 10;40(8):847-854. doi: 10.1200/JCO.21.01548. Epub 2021 Dec 10.
6
An international consortium proposal of uniform response criteria for myelodysplastic/myeloproliferative neoplasms (MDS/MPN) in adults.一份关于成人骨髓增生异常/骨髓增殖性肿瘤（MDS/MPN）统一反应标准的国际联盟提案。
Blood. 2015 Mar 19;125(12):1857-65. doi: 10.1182/blood-2014-10-607341. Epub 2015 Jan 26.
7
IDH2 mutation-induced histone and DNA hypermethylation is progressively reversed by small-molecule inhibition.异柠檬酸脱氢酶2（IDH2）突变诱导的组蛋白和DNA高甲基化可通过小分子抑制作用逐渐逆转。
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Cancer Cell. 2010 Dec 14;18(6):553-67. doi: 10.1016/j.ccr.2010.11.015. Epub 2010 Dec 9.