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伴有内部串联重复的急性髓系白血病患者造血干细胞移植的临床实践推荐:欧洲血液与骨髓移植学会急性白血病工作组的立场声明

Clinical practice recommendation on hematopoietic stem cell transplantation for acute myeloid leukemia patients with -internal tandem duplication: a position statement from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.

作者信息

Bazarbachi Ali, Bug Gesine, Baron Frederic, Brissot Eolia, Ciceri Fabio, Dalle Iman Abou, Döhner Hartmut, Esteve Jordi, Floisand Yngvar, Giebel Sebastian, Gilleece Maria, Gorin Norbert-Claude, Jabbour Elias, Aljurf Mahmoud, Kantarjian Hagop, Kharfan-Dabaja Mohamed, Labopin Myriam, Lanza Francesco, Malard Florent, Peric Zinaida, Prebet Thomas, Ravandi Farhad, Ruggeri Annalisa, Sanz Jaime, Schmid Christoph, Shouval Roni, Spyridonidis Alexandros, Versluis Jurjen, Vey Norbert, Savani Bipin N, Nagler Arnon, Mohty Mohamad

机构信息

Bone Marrow Transplantation Program, Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon

Department of Anatomy, Cell Biology, and Physiological Sciences, American University of Beirut, Beirut, Lebanon.

出版信息

Haematologica. 2020 Jun;105(6):1507-1516. doi: 10.3324/haematol.2019.243410. Epub 2020 Apr 2.

Abstract

The () gene is mutated in 25-30% of patients with acute myeloid leukemia (AML). Because of the poor prognosis associated with -internal tandem duplication mutated AML, allogeneic hematopoietic stem-cell transplantation (SCT) was commonly performed in first complete remission. Remarkable progress has been made in frontline treatments with the incorporation of FLT3 inhibitors and the development of highly sensitive minimal/measurable residual disease assays. Similarly, recent progress in allogeneic hematopoietic SCT includes improvement of transplant techniques, the use of haploidentical donors in patients lacking an HLA matched donor, and the introduction of FLT3 inhibitors as post-transplant maintenance therapy. Nevertheless, current transplant strategies vary between centers and differ in terms of transplant indications based on the internal tandem duplication allelic ratio and concomitant nucleophos-min-1 mutation, as well as in terms of post-transplant maintenance/consolidation. This review generated by international leukemia or transplant experts, mostly from the European Society for Blood and Marrow Transplantation, attempts to develop a position statement on best approaches for allogeneic hematopoietic SCT for AML with -internal tandem duplication including indications for and modalities of such transplants and on the potential optimization of post-transplant maintenance with FLT inhibitors.

摘要

在25%-30%的急性髓系白血病(AML)患者中,()基因发生突变。由于伴有FMS样酪氨酸激酶3(FLT3)-内部串联重复(ITD)突变的AML预后较差,异基因造血干细胞移植(SCT)通常在首次完全缓解时进行。随着FLT3抑制剂的引入以及高灵敏度微小/可测量残留病检测方法的发展,一线治疗取得了显著进展。同样,异基因造血SCT的最新进展包括移植技术的改进、在缺乏人类白细胞抗原(HLA)匹配供者的患者中使用单倍体相合供者,以及引入FLT3抑制剂作为移植后维持治疗。然而,目前各中心的移植策略各不相同,在基于ITD等位基因比例和伴发核仁磷酸蛋白1(NPM1)突变的移植适应证方面存在差异,在移植后维持/巩固方面也存在差异。这篇由国际白血病或移植专家撰写(大多来自欧洲血液与骨髓移植学会)的综述,试图就伴有FLT3-ITD的AML异基因造血SCT的最佳方法制定一份立场声明,包括此类移植的适应证和方式,以及关于使用FLT抑制剂进行移植后维持治疗的潜在优化。

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