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异体造血干细胞移植治疗镰状细胞病:原因、对象和方法。

Allogeneic hematopoietic stem cell transplant for sickle cell disease: The why, who, and what.

机构信息

Division of Hematology and Oncology, Department of Medicine, SUNY Downstate Health Sciences University, Brooklyn, NY 11203, United States.

Division of Hematology and Oncology, Department of Medicine, SUNY Downstate Health Sciences University, Brooklyn, NY 11203, United States.

出版信息

Blood Rev. 2021 Nov;50:100868. doi: 10.1016/j.blre.2021.100868. Epub 2021 Jul 18.

DOI:10.1016/j.blre.2021.100868
PMID:34332804
Abstract

Allogeneic hematopoietic stem cell transplants (allo-HSCTs) from matched-related donors (MRDs), mismatched-related donors (MMRDs), and matched-unrelated donors (MUDs) are increasingly being used to treat sickle cell disease (SCD) in both pediatric and adult patients. The overall results have been extremely encouraging, especially if a MRD is available and the transplant being performed before the age of 13. Although there is a general consensus that patients with high-risk SCD, even in adults and irrespective of donor characteristics, should be offered allo-HSCT, the debates on optimal patient selection and timing of transplant have yet to be resolved. Unlike patients with hematologic malignancies, there are also a number of clinical issues that require to be addressed in patients with SCD undergoing allo-HSCT. In this review, we will discuss the reasons allo-HSCT should be offered more widely to patients with SCD, the challenges facing physicians in patient selection and timing of transplant, and the awareness of and solutions to prevent the complications that are unique or more common in SCD undergoing allo-HSCT.

摘要

异基因造血干细胞移植(allo-HSCT)在儿童和成人患者中越来越多地用于治疗镰状细胞病(SCD),包括来自匹配相关供体(MRD)、不匹配相关供体(MMRD)和匹配无关供体(MUD)的移植。总体结果令人非常鼓舞,尤其是在可获得 MRD 且移植在 13 岁之前进行的情况下。尽管普遍认为应向高危 SCD 患者(即使是成人患者且不论供体特征如何)提供 allo-HSCT,但关于最佳患者选择和移植时机的争论尚未解决。与血液系统恶性肿瘤患者不同,SCD 患者在接受 allo-HSCT 时还存在许多需要解决的临床问题。在这篇综述中,我们将讨论向 SCD 患者更广泛地提供 allo-HSCT 的原因、医生在患者选择和移植时机方面面临的挑战,以及对 allo-HSCT 中 SCD 特有或更常见的并发症的认识和解决方案。

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引用本文的文献

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Beyond the traditional oncology patient: the role of palliative care in patients with sickle cell disease receiving stem cell transplantation or gene therapy.超越传统肿瘤患者:姑息治疗在接受干细胞移植或基因治疗的镰状细胞病患者中的作用。
Front Oncol. 2025 Feb 13;15:1535851. doi: 10.3389/fonc.2025.1535851. eCollection 2025.
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Biophysical chemistry behind sickle cell anemia and the mechanism of voxelotor action.镰状细胞贫血背后的生物物理化学和沃瑟罗特作用机制。
Sci Rep. 2024 Jan 22;14(1):1861. doi: 10.1038/s41598-024-52476-8.