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异基因造血细胞移植治疗骨髓纤维化:EBMT 慢性恶性肿瘤工作组关于移植物失败、移植物功能不良和复发的定义和管理策略建议:最佳实践推荐。

Allogeneic haematopoietic cell transplantation for myelofibrosis: proposed definitions and management strategies for graft failure, poor graft function and relapse: best practice recommendations of the EBMT Chronic Malignancies Working Party.

机构信息

Department of Haematology, Guy's and St. Thomas' NHS Foundation Trust and University College Hospitals, London, UK.

Hospital Clinico Universitario, Valencia, Spain.

出版信息

Leukemia. 2021 Sep;35(9):2445-2459. doi: 10.1038/s41375-021-01294-2. Epub 2021 May 26.

DOI:10.1038/s41375-021-01294-2
PMID:34040148
Abstract

Allogeneic haematopoietic cell transplantation (allo-HCT) remains the only curative approach in myelofibrosis (MF). Despite advances over recent decades, relapse and non-relapse mortality rates remain significant. Relapse rates vary between 15 and 25% across retrospective studies and management strategies vary widely, ranging from palliation to adoptive immunotherapy and, in some cases, a second allo-HCT. Moreover, in allo-HCT, there is a higher incidence of poor graft function and graft failure due to splenomegaly and a hostile "pro-inflammatory" marrow niche. The Practice Harmonisation and Guidelines subcommittee of the Chronic Malignancies Working Party (CMWP) of EBMT convened an international panel consisting of transplant haematologists, histopathologists and molecular biologists to propose practical, clinically relevant definitions of graft failure, poor graft function and relapse as well as management strategies following allo-HCT. A systematic approach to molecular monitoring, histopathological assessment and chimerism testing is proposed. These proposed recommendations aim to increase the accuracy and uniformity of reporting and to thereby facilitate the development of more consistent approaches to these challenging issues. In addition, we propose management strategies for these complications.

摘要

异基因造血细胞移植(allo-HCT)仍然是骨髓纤维化(MF)的唯一治愈方法。尽管近几十年来取得了进展,但复发和非复发死亡率仍然很高。在回顾性研究中,复发率在 15%至 25%之间变化,管理策略差异很大,从姑息治疗到过继免疫治疗,在某些情况下还需要进行第二次 allo-HCT。此外,在 allo-HCT 中,由于脾肿大和敌对的“促炎”骨髓微环境,嵌合率低和移植物功能不良的发生率更高。EBMT 的慢性恶性肿瘤工作组(CMWP)的实践协调和指南小组委员会召集了一个由移植血液学家、组织病理学家和分子生物学家组成的国际小组,提出了实用的、与临床相关的嵌合率低、移植物功能不良和复发的定义以及 allo-HCT 后的管理策略。提出了一种系统的分子监测、组织病理学评估和嵌合体检测方法。这些建议旨在提高报告的准确性和一致性,从而促进对这些具有挑战性问题的更一致方法的发展。此外,我们还提出了这些并发症的管理策略。

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本文引用的文献

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Genomic analysis of primary and secondary myelofibrosis redefines the prognostic impact of ASXL1 mutations: a FIM study.原发性和继发性骨髓纤维化的基因组分析重新定义了 ASXL1 突变的预后影响:FIM 研究。
Blood Adv. 2021 Mar 9;5(5):1442-1451. doi: 10.1182/bloodadvances.2020003444.
2
Impact of spleen size and splenectomy on outcomes of allogeneic hematopoietic cell transplantation for myelofibrosis: A retrospective analysis by the chronic malignancies working party on behalf of European society for blood and marrow transplantation (EBMT).脾大和脾切除对骨髓纤维化患者异基因造血细胞移植结局的影响:慢性恶性肿瘤工作组代表欧洲血液和骨髓移植学会(EBMT)的回顾性分析。
Am J Hematol. 2021 Jan;96(1):69-79. doi: 10.1002/ajh.26020. Epub 2020 Oct 27.
3
[Selected donor CD34(+) cell boosts for salvage treatment of poor graft function following allogeneic hematopoietic stem cell transplantation in primary myelofibrosis: 3 cases report].
[选择供体CD34(+)细胞增强剂用于原发性骨髓纤维化异基因造血干细胞移植后移植物功能不良的挽救治疗:3例报告]
Zhonghua Xue Ye Xue Za Zhi. 2024 Aug 14;45(8):785-788. doi: 10.3760/cma.j.cn121090-20240117-00029.
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Myelofibrosis and allogeneic transplantation: critical points and challenges.骨髓纤维化与异基因移植:关键点与挑战
Front Oncol. 2024 Jun 20;14:1396435. doi: 10.3389/fonc.2024.1396435. eCollection 2024.
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Decision analysis for transplant candidates with primary myelofibrosis in the ruxolitinib era.原发性骨髓纤维化患者在芦可替尼时代的移植决策分析。
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Bone Marrow Transplant. 2024 Jul;59(7):997-1005. doi: 10.1038/s41409-024-02266-7. Epub 2024 Apr 5.
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STAT5a and SH2B3 novel mutations display malignancy roles in a triple-negative primary myelofibrosis patient.STAT5a和SH2B3新突变在一名三阴性原发性骨髓纤维化患者中显示出恶性作用。
Cancer Gene Ther. 2024 Mar;31(3):484-494. doi: 10.1038/s41417-023-00719-7. Epub 2023 Dec 22.
Incidence and Outcome of Late Relapse after Allogeneic Stem Cell Transplantation for Myelofibrosis.骨髓纤维化异基因造血干细胞移植后晚期复发的发生率和结果。
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Biol Blood Marrow Transplant. 2019 Nov;25(11):2167-2171. doi: 10.1016/j.bbmt.2019.06.034. Epub 2019 Jul 5.
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Incidence, Risk Factors, and Outcomes of Primary Poor Graft Function after Allogeneic Hematopoietic Stem Cell Transplantation.异基因造血干细胞移植后原发性移植物功能不良的发生率、危险因素和结局。
Biol Blood Marrow Transplant. 2019 Sep;25(9):1898-1907. doi: 10.1016/j.bbmt.2019.05.036. Epub 2019 Jun 6.