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阿扎胞苷、恩西地平与维奈克拉联合治疗原发性难治性急性髓系白血病的病例报告

Case report of combination therapy with Azacytidine, Enasidenib and Venetoclax in primary refractory AML.

作者信息

Jasra Sakshi, Kazemi Mohammed, Shah Nishi, Chen Jiahao, Fehn Karen, Wang Yanhua, Mantzaris Ioannis, Kornblum Noah, Sica Alejandro, Bachier LizaMarie, Goldfinger Mendel, Gritsman Kira, Braunschweig Ira, Steidl Ulrich, Shastri Aditi, Verma Amit

机构信息

Department of Hematology/Oncology, University of Vermont, 89 Beaumont Avenue, Given E-214, Burlington, VT, 05405, USA.

Albert Einstein College of Medicine, Department of Hematology/Oncology, Bronx, NY, USA.

出版信息

Exp Hematol Oncol. 2021 Jan 4;10(1):1. doi: 10.1186/s40164-020-00186-y.

DOI:10.1186/s40164-020-00186-y
PMID:33397455
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7784272/
Abstract

Optimal treatment of acute myeloid leukemia (AML) arising in elderly patients remains a challenge. FDA approval of Ivosidenib and Enasidenib, small molecule inhibitors of isocitrate dehydrogenase enzymes (IDH1 and 2) have opened new avenues of treatment. We present a 60-year-old woman with refractory AML, achieving complete response to the combination therapy of hypomethylating agent, Azacytidine with the IDH2 inhibitor, Enasidenib, and BCL2 inhibitor, Venetoclax. To our knowledge, this is the first case report of a patient with IDH2 mutated refractory AML achieving complete response to combination therapy with azacytidine, enasidenib and venetoclax.

摘要

老年患者急性髓系白血病(AML)的最佳治疗仍然是一项挑战。美国食品药品监督管理局(FDA)批准的异柠檬酸脱氢酶(IDH1和2)小分子抑制剂艾伏尼布和恩杂鲁胺开辟了新的治疗途径。我们报告了一名60岁的难治性AML女性患者,其对去甲基化药物阿扎胞苷与IDH2抑制剂恩杂鲁胺以及BCL2抑制剂维奈克拉的联合治疗实现了完全缓解。据我们所知,这是首例IDH2突变的难治性AML患者对阿扎胞苷、恩杂鲁胺和维奈克拉联合治疗实现完全缓解的病例报告。

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本文引用的文献

1
Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia.阿扎胞苷和维奈托克治疗未经治急性髓系白血病。
N Engl J Med. 2020 Aug 13;383(7):617-629. doi: 10.1056/NEJMoa2012971.
2
Differentiation Syndrome with Ivosidenib and Enasidenib Treatment in Patients with Relapsed or Refractory IDH-Mutated AML: A U.S. Food and Drug Administration Systematic Analysis.伊维菌素和依维莫司治疗复发性或难治性 IDH 突变型 AML 患者的分化综合征:美国食品和药物管理局系统分析。
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Myelodysplastic syndrome progression to acute myeloid leukemia at the stem cell level.
急性髓系白血病中的2-羟基戊二酸:从发病机制到治疗的历程
Biomedicines. 2022 Jun 9;10(6):1359. doi: 10.3390/biomedicines10061359.
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Older Patients with Acute Myeloid Leukemia Deserve Individualized Treatment.老年急性髓系白血病患者应接受个体化治疗。
Curr Oncol Rep. 2022 Nov;24(11):1387-1400. doi: 10.1007/s11912-022-01299-9. Epub 2022 Jun 2.
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Novel agents and regimens for hematological malignancies: recent updates from 2020 ASH annual meeting.血液恶性肿瘤的新型药物和治疗方案:2020 年美国血液学会年会最新进展。
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骨髓增生异常综合征向急性髓系白血病在干细胞水平的进展。
Nat Med. 2019 Jan;25(1):103-110. doi: 10.1038/s41591-018-0267-4. Epub 2018 Dec 3.
4
Durable Remissions with Ivosidenib in IDH1-Mutated Relapsed or Refractory AML.ivosidenib 治疗 IDH1 突变复发性或难治性 AML 的持久缓解。
N Engl J Med. 2018 Jun 21;378(25):2386-2398. doi: 10.1056/NEJMoa1716984. Epub 2018 Jun 2.
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Clinical experience with the BCL2-inhibitor venetoclax in combination therapy for relapsed and refractory acute myeloid leukemia and related myeloid malignancies. Venetoclax 联合治疗复发/难治性急性髓系白血病及相关髓系恶性肿瘤的临床经验。
Am J Hematol. 2018 Mar;93(3):401-407. doi: 10.1002/ajh.25000. Epub 2017 Dec 23.
6
Enasidenib in mutant relapsed or refractory acute myeloid leukemia.恩杂鲁胺用于治疗突变型复发或难治性急性髓系白血病。 (注:原文中药物名可能有误,推测正确药物名应该是Enasidenib为恩杂鲁胺,而这里治疗白血病的应该是Enasentinib,中文名为恩西地平 ,以下按照正确药物名给出译文) 恩西地平用于治疗突变型复发或难治性急性髓系白血病。
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Outcomes after induction chemotherapy in patients with acute myeloid leukemia arising from myelodysplastic syndrome.骨髓增生异常综合征相关急性髓系白血病患者接受诱导化疗后的结果。
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Leukemic IDH1 and IDH2 mutations result in a hypermethylation phenotype, disrupt TET2 function, and impair hematopoietic differentiation.白血病 IDH1 和 IDH2 突变导致超甲基化表型,破坏 TET2 功能,并损害造血分化。
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