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急性淋巴细胞白血病的干细胞移植:你总会有供者,为什么不总是使用?

英文原题:Stem cell transplantation for ALL: you've always got a donor, why not always use it?

查看英文原题

Stem cell transplantation for ALL: you've always got a donor, why not always use it?

PubMed 2023/12/08(内容时间) Hematology Am Soc Hematol Educ Program Q1 · IF 3.7(JCR 2025)

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中文摘要

造血干细胞移植(HSCT)是高危儿童急性淋巴细胞白血病(ALL)的成熟治疗策略,可带来治愈机会。本文探讨在合适供者普遍可及的现代背景下,是否应更普遍地将HSCT用于儿童ALL。尽管化疗、靶向治疗和免疫治疗取得重大进展,一部分具有高危特征或复发的儿童ALL患者预后仍差。对于这部分患者,HSCT通常仍是唯一具有潜在治愈性的措施,可利用移植物抗白血病效应实现长期疾病控制。然而,移植流程复杂且伴随风险,传统上限制了其广泛应用。随着HLA配型改善、替代供者来源增加、预处理方案毒性降低及支持治疗改进,情况正在变化。同时,CD19 CAR-T 等新兴疗法也为复发/难治ALL的最终治疗选择带来新考量。本文回顾当前关键证据及HSCT能否更普遍用于ALL的争论,并在干细胞供者持续可及的背景下重新评估传统治疗分层。

展开英文摘要原文

Hematopoietic stem cell transplantation (HSCT) represents a consolidated therapeutic strategy for high-risk pediatric acute lymphoblastic leukemia (ALL), offering the potential for curative treatment. This manuscript delves into the debate around the more universal application of HSCT for pediatric ALL in the modern era, considering the ubiquitous availability of suitable donors.

In fact, despite significant advancements in chemotherapy, targeted therapy, and immunotherapy, a subset of pediatric patients with ALL with high-risk features or relapse continue to encounter poor prognostic outcomes. For this subgroup of patients, HSCT often remains the only potentially curative measure, leveraging the graft-versus- leukemia effect for long-term disease control. Nevertheless, the procedure's complexity and associated risks have traditionally curtailed its widespread use.

However, the scenario is shifting with improvements in HLA matching, availability of alternative donor sources, less toxic conditioning regimens, and improved supportive care protocols. Concurrently, emerging therapies like CD19+ CAR T cells present new considerations for definitive therapy selection in relapsed/ refractory ALL. This article reviews critical current evidence and debates the potential of HSCT as a more universal treatment for ALL, reevaluating traditional treatment stratification in light of the constant availability of stem cell donors.

论文信息

作者
Shyr D、Davis KL、Bertaina A
单位
Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA.
文献类型
综述
期刊
Hematology. American Society of Hematology. Education Program2023 Dec 8
原文标识
PubMed 38066901 · DOI 10.1182/hematology.2023000423