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Azacytidine Monotherapy in an Infant With Mosaic NRAS-Driven Juvenile Myelomonocytic Leukemia.

作者信息

Grenier Jeremy M, Tasian Sarah K, Stieglitz Elliot, Abdullaev Zied, Wertheim Gerald B, Li Marilyn M, Phillips Charles A

机构信息

Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA.

出版信息

Pediatr Blood Cancer. 2025 May;72(5):e31647. doi: 10.1002/pbc.31647. Epub 2025 Mar 4.

DOI:10.1002/pbc.31647
PMID:40040253
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11970265/
Abstract
摘要

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本文引用的文献

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Efficacy of the Allosteric MEK Inhibitor Trametinib in Relapsed and Refractory Juvenile Myelomonocytic Leukemia: a Report from the Children's Oncology Group.变构 MEK 抑制剂曲美替尼治疗复发/难治性幼年髓单核细胞白血病的疗效：来自儿童肿瘤协作组的报告。
Cancer Discov. 2024 Sep 4;14(9):1590-1598. doi: 10.1158/2159-8290.CD-23-1376.
2
Clinical responses in pediatric patients with relapsed/refractory leukemia treated with azacitidine and venetoclax.接受阿扎胞苷和维奈克拉治疗的复发/难治性白血病儿科患者的临床反应
Haematologica. 2023 Nov 1;108(11):3142-3147. doi: 10.3324/haematol.2022.282637.
3
Juvenile myelomonocytic leukemia in the molecular era: a clinician's guide to diagnosis, risk stratification, and treatment.分子时代的青少年骨髓单核细胞白血病：诊断、风险分层和治疗的临床医生指南。
Blood Adv. 2021 Nov 23;5(22):4783-4793. doi: 10.1182/bloodadvances.2021005117.
4
Response to upfront azacitidine in juvenile myelomonocytic leukemia in the AZA-JMML-001 trial.AZA-JMML-001 试验中 upfront 阿扎胞苷治疗幼年型粒单核细胞白血病的疗效。
Blood Adv. 2021 Jul 27;5(14):2901-2908. doi: 10.1182/bloodadvances.2020004144.
5
Molecular and phenotypic diversity of CBL-mutated juvenile myelomonocytic leukemia.CBL 突变型幼年骨髓单核细胞白血病的分子和表型多样性。
Haematologica. 2022 Jan 1;107(1):178-186. doi: 10.3324/haematol.2020.270595.
6
International Consensus Definition of DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia.国际共识定义青少年骨髓单核细胞白血病中的 DNA 甲基化亚群。
Clin Cancer Res. 2021 Jan 1;27(1):158-168. doi: 10.1158/1078-0432.CCR-20-3184. Epub 2020 Nov 2.
7
Development and Clinical Validation of a Large Fusion Gene Panel for Pediatric Cancers.开发和临床验证用于儿科癌症的大型融合基因panel。
J Mol Diagn. 2019 Sep;21(5):873-883. doi: 10.1016/j.jmoldx.2019.05.006. Epub 2019 Jun 27.
8
Clinical utility of custom-designed NGS panel testing in pediatric tumors.定制化 NGS -panel 检测在儿科肿瘤中的临床实用性。
Genome Med. 2019 May 28;11(1):32. doi: 10.1186/s13073-019-0644-8.
9
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Blood. 2019 Mar 7;133(10):1060-1070. doi: 10.1182/blood-2018-11-844688. Epub 2019 Jan 22.
10
JMML genomics and decisions.JMML 的基因组学与决策。
Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):307-312. doi: 10.1182/asheducation-2018.1.307.