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成人血液系统疾病造血细胞移植(HCT)的长期结果——一项系统评价

Long-Term Results of Hematopoietic Cell Transplantation (HCT) for Adults with Blood Disorders- A Systematic Review.

作者信息

Karaszewski Kajetan, Góra Jakub, Ploch Weronika, Jędrzejczak Wiesław Wiktor

机构信息

Department of Hematology, Transplantation and Internal Medicine, Medical University of Warsaw, ul. Banacha 1A, 02-097, Warsaw, Poland.

Doctoral School of the Medical University of Warsaw, ul. Żwirki I Wigury 61, 02-091, Warsaw, Poland.

出版信息

Stem Cell Rev Rep. 2025 Jun 18. doi: 10.1007/s12015-025-10912-w.

DOI:10.1007/s12015-025-10912-w
PMID:40531405
Abstract

Both autologous and allogeneic stem cell transplantations are widely used as a standard of care or significant clinical option in treating various malignant and non-malignant diseases of the hematopoietic system. The value of these treatment methods would be better assessed through the review of long-term progression-free and overall survival (PFS and OS) rates, which are highly dependent on the primary diagnosis and stage of disease at the time of transplantation as well as on the specific type of used transplantation procedure. While the best (over 90%) 5-, 10- and 15-year survival rates are achieved in children with genetic errors after HLA-matched allogeneic transplantation, in adults these results are worse. Intermediate results (50-80%) of 5- and 10-year survival rates are obtained in malignancies transplanted in complete remission, and the worst (between 10 and 30%) 5- and 10-year survival rates are seen in malignancies transplanted without remission. However, none of the other treatment methods in these latter situations allows to obtain any long-term survival. In conclusion, transplantation of hematopoietic cells is a group of therapeutic methods with unprecedented effectiveness in reversing outcome of deadly disorders of the hematopoietic system. Moreover, its therapeutic effect is usually maintained for 10 years or more.

摘要

自体和异体干细胞移植都被广泛用作治疗各种造血系统恶性和非恶性疾病的标准治疗方法或重要临床选择。通过回顾长期无进展生存率和总生存率(PFS和OS)可以更好地评估这些治疗方法的价值,而这高度依赖于移植时疾病的初始诊断和分期以及所采用移植程序的具体类型。虽然在 HLA 匹配的异体移植后,患有基因缺陷的儿童能取得最佳的(超过90%)5年、10年和15年生存率,但在成人中这些结果较差。在完全缓解期进行移植的恶性肿瘤患者可获得5年和10年生存率的中等结果(50 - 80%),而在未缓解状态下进行移植的恶性肿瘤患者的5年和10年生存率最差(10%至30%)。然而,在这些后一种情况下,其他任何治疗方法都无法实现长期生存。总之,造血细胞移植是一组在逆转造血系统致命疾病结局方面具有前所未有的疗效的治疗方法。此外,其治疗效果通常能维持10年或更长时间。

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本文引用的文献

1
Impact of hematopoietic cell transplantation and quizartinib in newly diagnosed patients with acute myeloid leukemia and FMS-like tyrosine kinase 3-internal tandem duplications in the QuANTUM-First trial.在QuANTUM-First试验中,造血细胞移植和quizartinib对新诊断的急性髓系白血病且伴有FMS样酪氨酸激酶3内部串联重复的患者的影响。
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Daratumumab with lenalidomide as maintenance after transplant in newly diagnosed multiple myeloma: the AURIGA study.达雷妥尤单抗联合来那度胺用于新诊断多发性骨髓瘤移植后维持治疗:AURIGA研究
Blood. 2025 Jan 16;145(3):300-310. doi: 10.1182/blood.2024025746.
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Global characteristics and outcomes of autologous hematopoietic stem cell transplantation for newly diagnosed multiple myeloma: A study of the worldwide network for blood and marrow transplantation (WBMT).
自体造血干细胞移植治疗初诊多发性骨髓瘤的全球特征和结局:全球血液和骨髓移植网络(WBMT)的研究。
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Sorafenib maintenance in FLT3-ITD mutated AML after allogeneic HCT: a real-world, single-center experience.异基因造血干细胞移植后索拉非尼维持治疗FLT3-ITD突变的急性髓系白血病:一项真实世界、单中心经验。
Front Oncol. 2024 Jun 24;14:1391743. doi: 10.3389/fonc.2024.1391743. eCollection 2024.
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Hematopoietic stem cell transplantation for DLBCL: a report from the European Society for Blood and Marrow Transplantation on more than 40,000 patients over 32 years.造血干细胞移植治疗弥漫性大 B 细胞淋巴瘤:欧洲血液和骨髓移植学会 32 年超过 40000 例患者的报告。
Blood Cancer J. 2024 Jul 5;14(1):106. doi: 10.1038/s41408-024-01085-9.
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Current Trends and Outcomes in Cellular Therapy Activity in the United States, Including Prospective Patient-Reported Outcomes Data Collection in the Center for International Blood and Marrow Transplant Research Registry.美国细胞治疗活动的当前趋势与成果,包括国际血液和骨髓移植研究中心登记处对患者前瞻性报告结果的数据收集。
Transplant Cell Ther. 2024 Sep;30(9):917.e1-917.e12. doi: 10.1016/j.jtct.2024.06.021. Epub 2024 Jun 27.
7
Tandem autologous hematopoietic stem cell transplantation for patients with multiple myeloma: a systematic review and meta-analysis.自体造血干细胞移植治疗多发性骨髓瘤患者的系统评价和荟萃分析。
Hematology. 2024 Dec;29(1):2343164. doi: 10.1080/16078454.2024.2343164. Epub 2024 Apr 23.
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