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芦可替尼在一名移植后类固醇难治性移植物抗宿主病的急性髓系白血病患者中的长期应用

Long-Term Use of Ruxolitinib in an AML Patient with Posttransplant Steroid Refractory GVHD.

作者信息

Sobash Philip T, Guddati Achuta K, Kota Vamsi

机构信息

Internal Medicine, White River Health System, Batesville, AR, USA.

Medical Oncology, Augusta University, Augusta, GA, USA.

出版信息

Case Rep Oncol Med. 2020 Apr 6;2020:4936846. doi: 10.1155/2020/4936846. eCollection 2020.

DOI:10.1155/2020/4936846
PMID:32318300
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7166298/
Abstract

Ruxolitinib has become a new therapeutic option for steroid refractory graft-versus-host disease (srGVHD), with a substantial remission rate. Its anti-inflammatory properties by blocking interleukin pathways have made it a novel therapeutic approach to inflammatory disease processes, such as GVHD. The long-term use of ruxolitinib has not been explicitly studied outside the context in the treatment of multiple myeloma. With current clinical trials underway for the use of ruxolitinib in srGVHD, there are still no current guidelines or protocols for long-term clinical use. Of the available literature showing ruxolitinib utilization for srGVHD, most cases lead to resolution and eventual discontinuation. We present a case of a 32-year-old male on ruxolitinib with GVHD status postmatched unrelated donor stem cell transplant (MUD SCT) for acute myeloid leukemia (AML) with FLT3 mutation currently on ruxolitinib for 5 years who is not able to tolerate reduction in dosage due to flare-ups. We discuss the clinical implications and nuance of therapy with ruxolitinib with unknown long-term effects and weigh the risks and benefits.

摘要

鲁索替尼已成为类固醇难治性移植物抗宿主病(srGVHD)的一种新的治疗选择,缓解率颇高。其通过阻断白细胞介素通路发挥的抗炎特性,使其成为治疗诸如GVHD等炎症性疾病过程的一种新型治疗方法。在多发性骨髓瘤治疗背景之外,尚未对鲁索替尼的长期使用进行明确研究。鉴于目前正在进行鲁索替尼用于srGVHD的临床试验,目前仍没有长期临床使用的指南或方案。在现有显示鲁索替尼用于srGVHD的文献中,大多数病例病情得到缓解并最终停药。我们报告一例32岁男性,因急性髓系白血病(AML)伴FLT3突变接受了匹配无关供体干细胞移植(MUD SCT),目前使用鲁索替尼治疗GVHD已达5年,因病情突然发作无法耐受剂量减少。我们讨论了鲁索替尼治疗的临床意义及细微差别,其长期影响未知,并权衡了风险与益处。

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Long-Term Use of Ruxolitinib in an AML Patient with Posttransplant Steroid Refractory GVHD.芦可替尼在一名移植后类固醇难治性移植物抗宿主病的急性髓系白血病患者中的长期应用
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本文引用的文献

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Efficacy, Toxicity, and Infectious Complications in Ruxolitinib-Treated Patients with Corticosteroid-Refractory Graft-versus-Host Disease after Hematopoietic Cell Transplantation.造血细胞移植后糖皮质激素难治性移植物抗宿主病患者接受芦可替尼治疗的疗效、毒性和感染并发症。
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Ruxolitinib treatment for steroid refractory acute and chronic graft vs host disease in children: Clinical and immunological results.芦可替尼治疗儿童激素耐药性急性和慢性移植物抗宿主病:临床和免疫学结果。
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Ruxolitinib in GvHD (RIG) study: a multicenter, randomized phase 2 trial to determine the response rate of Ruxolitinib and best available treatment (BAT) versus BAT in steroid-refractory acute graft-versus-host disease (aGvHD) (NCT02396628).Ruxolitinib 在移植物抗宿主病(GvHD)中的研究(RIG):一项多中心、随机的 2 期临床试验,旨在确定 Ruxolitinib 和最佳可用治疗(BAT)在激素难治性急性移植物抗宿主病(aGvHD)中的反应率,与 BAT 相比(NCT02396628)。
BMC Cancer. 2018 Nov 19;18(1):1132. doi: 10.1186/s12885-018-5045-7.
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Ruxolitinib: a steroid sparing agent in chronic graft-versus-host disease.芦可替尼:慢性移植物抗宿主病的类固醇节约剂。
Bone Marrow Transplant. 2018 Jul;53(7):826-831. doi: 10.1038/s41409-017-0081-5. Epub 2018 Jan 24.
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Ruxolitinib for the treatment of patients with steroid-refractory GVHD: an introduction to the REACH  trials.芦可替尼用于治疗类固醇难治性移植物抗宿主病患者:REACH试验介绍
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The role of JAK-STAT signaling pathway and its regulators in the fate of T helper cells.JAK-STAT信号通路及其调节因子在辅助性T细胞命运中的作用。
Cell Commun Signal. 2017 Jun 21;15(1):23. doi: 10.1186/s12964-017-0177-y.
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Ruxolitinib in corticosteroid-refractory graft-versus-host disease after allogeneic stem cell transplantation: a multicenter survey.芦可替尼用于异基因干细胞移植后对皮质类固醇难治的移植物抗宿主病:一项多中心调查
Leukemia. 2015 Oct;29(10):2062-8. doi: 10.1038/leu.2015.212. Epub 2015 Jul 31.
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First- and second-line systemic treatment of acute graft-versus-host disease: recommendations of the American Society of Blood and Marrow Transplantation.急性移植物抗宿主病的一线和二线全身治疗:美国血液和骨髓移植学会的建议。
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