HCT 在不断扩大的先天性免疫缺陷中的应用。

IF 3.1 3区 医学 Q2 HEMATOLOGY
Current Opinion in Hematology Pub Date : 2025-01-01 Epub Date: 2024-11-25 DOI:10.1097/MOH.0000000000000849
Rafaella Muratori, Carmem Bonfim
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引用次数: 0

摘要

综述目的:自1968年移植首次用于治疗严重联合免疫缺陷症(SCID)以来,造血干细胞移植(HSCT)与先天性免疫错误(IEI)一直密切相关。从那时起,不断有新的基因和疾病被列入先天性免疫缺陷名单,有关适应症和治疗效果的新数据也不断涌现。我们回顾了造血细胞移植(HCT)治疗 IEI 领域的最新数据和进展,包括新的疾病和并发症:最近的发现:从单倍体移植、新生儿筛查结果和多中心研究中获得的新数据显示,IEI 的治疗效果很好。免疫调节失调疾病在疾病控制方面值得特别关注,可能需要在移植前使用额外的药物。X连锁慢性肉芽肿女性携带者可能表现出严重的表型,需要进行造血干细胞移植。从感染并发症和长期合并症中获得的启示应有助于指导治疗 IEI 患者的决策:从传统的适应症到最近描述的疾病,用于免疫缺陷的 HCT 是一个快速发展的领域。有关替代供体移植的新数据、大型队列的结果以及长期并发症为临床实践提供了宝贵的知识。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
HCT in the widening spectrum of congenital immunodeficiencies.

Purpose of review: Hematopoietic stem cell transplantation (HSCT) and inborn errors of immunity (IEI) have been closely linked since transplantation was first used to cure severe combined immunodeficiency (SCID) in 1968. Since then, novel genes and diseases have been continually added to the ongoing list of IEI, and new data on indications and outcomes have emerged. We review recent data and progress in the field of hematopoietic cell transplantation (HCT) for IEI including new diseases and complications.

Recent findings: Emerging data from haploidentical transplants, newborn screening results, and multicentric studies reveals promising outcomes for IEI. Immune dysregulation diseases deserve special attention regarding disease control and may require additional drugs pretransplant. Female carriers of X-linked Chronic granulomatous may present with a severe phenotype warranting the need for HCT. Insights from infectious complications and long-term comorbidities should help guide decisions to treat IEI patients.

Summary: From classical indications to recently described diseases, HCT for immunodeficiencies is a rapidly growing field. Novel data regarding alternative donor transplants, results from large cohorts, and long-term complications provide valuable knowledge for clinical practice.

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来源期刊
CiteScore
6.60
自引率
3.10%
发文量
78
审稿时长
6-12 weeks
期刊介绍: ​​​​​​​​Current Opinion in Hematology is an easy-to-digest bimonthly journal covering the most interesting and important advances in the field of hematology. Its hand-picked selection of editors ensure the highest quality selection of unbiased review articles on themes from nine key subject areas, including myeloid biology, Vascular biology, hematopoiesis and erythroid system and its diseases.
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