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Combined use of emapalumab and ruxolitinib in a patient with refractory hemophagocytic lymphohistiocytosis was safe and effective.

作者信息

Triebwasser Michael P, Barrett David M, Bassiri Hamid, Bunin Nancy, Elgarten Caitlin, Freedman Jason, Geera Abdallah S, Monos Dimitri, Lambert Michele P, Olson Timothy, Seif Alix E, Paessler Michele, Petrosa Whitney, Reilly Anne F, Romberg Neil, Sullivan Kathleen E, Quinn Ghazal Z, Behrens Edward, Teachey David T

机构信息

Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

出版信息

Pediatr Blood Cancer. 2021 Jul;68(7):e29026. doi: 10.1002/pbc.29026. Epub 2021 Mar 22.

DOI:10.1002/pbc.29026
PMID:33754483
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9269994/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f4db/9269994/f7d218664628/nihms-1815111-f0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f4db/9269994/f7d218664628/nihms-1815111-f0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f4db/9269994/f7d218664628/nihms-1815111-f0001.jpg

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本文引用的文献

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Human Inborn Errors of Immunity: 2019 Update of the IUIS Phenotypical Classification.人类先天性免疫缺陷:2019 年国际免疫学会联合会表型分类更新。
J Clin Immunol. 2020 Jan;40(1):66-81. doi: 10.1007/s10875-020-00758-x. Epub 2020 Feb 11.
2
Ruxolitinib in adult patients with secondary haemophagocytic lymphohistiocytosis: an open-label, single-centre, pilot trial.芦可替尼治疗成人继发性噬血细胞性淋巴组织细胞增生症:一项开放标签、单中心的试点试验。
Lancet Haematol. 2019 Dec;6(12):e630-e637. doi: 10.1016/S2352-3026(19)30156-5. Epub 2019 Sep 16.
3
Ruxolitinib for refractory/relapsed hemophagocytic lymphohistiocytosis.
Haematologica. 2024 Sep 1;109(9):2998-3003. doi: 10.3324/haematol.2023.284179.
4
Early hematopoietic cell transplantation for familial hemophagocytic lymphohistiocytosis in a regional treatment network in Japan.在日本的区域治疗网络中,对家族性噬血细胞性淋巴组织细胞增多症进行早期造血细胞移植。
Int J Hematol. 2024 May;119(5):592-602. doi: 10.1007/s12185-024-03721-3. Epub 2024 Mar 20.
5
HLH as an additional warning sign of inborn errors of immunity beyond familial-HLH in children: a systematic review.HLH 作为儿童先天免疫缺陷以外家族性 HLH 的附加预警信号:系统评价。
Front Immunol. 2024 Feb 13;15:1282804. doi: 10.3389/fimmu.2024.1282804. eCollection 2024.
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Emapalumab for the treatment of refractory cytokine release syndrome in pediatric patients.依马帕鲁单抗用于治疗儿科患者难治性细胞因子释放综合征。
Blood Adv. 2023 Sep 26;7(18):5603-5607. doi: 10.1182/bloodadvances.2023010712.
7
Advances in attractive therapeutic approach for macrophage activation syndrome in COVID-19.COVID-19 中巨噬细胞活化综合征有吸引力的治疗方法进展。
Front Immunol. 2023 Jul 6;14:1200289. doi: 10.3389/fimmu.2023.1200289. eCollection 2023.
8
Cellular and transcriptional impacts of Janus kinase and/or IFN-gamma inhibition in a mouse model of primary hemophagocytic lymphohistiocytosis.Janus 激酶和/或 IFN-γ抑制在原发性噬血细胞性淋巴组织细胞增多症小鼠模型中的细胞和转录影响。
Front Immunol. 2023 Apr 27;14:1137037. doi: 10.3389/fimmu.2023.1137037. eCollection 2023.
9
Efficacy and safety of emapalumab in macrophage activation syndrome.埃马鲁单抗治疗巨噬细胞活化综合征的疗效和安全性。
Ann Rheum Dis. 2023 Jun;82(6):857-865. doi: 10.1136/ard-2022-223739. Epub 2023 Mar 31.
10
Case report: Optimized ruxolitinib-based therapy in an infant with familial hemophagocytic lymphohistiocytosis type 3.病例报告:优化基于鲁索利替尼的治疗方案在家族性噬血细胞性淋巴组织细胞增多症 3 型婴儿中的应用。
Front Immunol. 2022 Nov 23;13:977463. doi: 10.3389/fimmu.2022.977463. eCollection 2022.
芦可替尼用于难治性/复发性噬血细胞性淋巴组织细胞增生症
Haematologica. 2020 May;105(5):e210-e212. doi: 10.3324/haematol.2019.222471. Epub 2019 Sep 12.
4
Role of interferon-γ in immune-mediated graft failure after allogeneic hematopoietic stem cell transplantation.干扰素-γ 在异基因造血干细胞移植后免疫介导的移植物失败中的作用。
Haematologica. 2019 Nov;104(11):2314-2323. doi: 10.3324/haematol.2019.216101. Epub 2019 Feb 21.
5
IFN-gamma mediates the rejection of haematopoietic stem cells in IFN-gammaR1-deficient hosts.γ干扰素在γ干扰素受体1缺陷宿主中介导造血干细胞的排斥反应。
PLoS Med. 2008 Jan 29;5(1):e26. doi: 10.1371/journal.pmed.0050026.
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IFN-gamma negatively modulates self-renewal of repopulating human hemopoietic stem cells.干扰素-γ对人类造血干细胞的自我更新起负向调节作用。
J Immunol. 2005 Jan 15;174(2):752-7. doi: 10.4049/jimmunol.174.2.752.