Abstract：
This study aims to perform an extensive review of the literature that evaluates various factors that affect the survival rates of patients with severe combined immunodeficiency (SCID) after hematopoietic stem cell transplantation (HSCT) in developed and developing countries. An extensive search of the literature was made in four different databases (PubMed, Embase, Scopus, and Web of Science). The search was carried out in December 2022 and updated in July 2023, and the terms such as \"hematopoietic stem cell transplantation,\" \"bone marrow transplant,\" \"mortality,\" \"opportunistic infections,\" and \"survival\" associated with \"severe combined immunodeficiency\" were sought based on the MeSH terms. The language of the articles was \"English,\" and only articles published from 2000 onwards were selected. Twenty-three articles fulfilled the inclusion criteria for review and data extraction. The data collected corroborates that early HSCT, but above all, HSCT in patients without active infections, is related to better overall survival. The universal implementation of newborn screening for SCID will be a fundamental pillar for enabling most transplants to be carried out in this \"ideal scenario\" at an early age and free from infection. HSCT with an HLA-identical sibling donor is also associated with better survival rates, but this is the least common scenario. For this reason, transplantation with matched unrelated donors (MUD) and mismatched related donors (mMRD/Haploidentical) appear as alternatives. The results obtained with MUD are improving and show survival rates similar to those of MSD, as well as they do not require manipulation of the graft with expensive technologies. However, they still have high rates of complications after HSCT. Transplants with mMRD/Haplo are performed just in a few large centers because of the high costs of the technology to perform CD3/CD19 depletion and TCRαβ/CD19 depletion or CD34 + selection techniques in vitro. The new possibility of in vivo T cell depletion using post-transplant cyclophosphamide could also be a viable alternative for performing mMRD transplants in centers that do not have this technology, especially in developing countries.
摘要：
这项研究旨在对文献进行广泛的回顾，以评估影响发达国家和发展中国家造血干细胞移植（HSCT）后严重联合免疫缺陷（SCID）患者生存率的各种因素。在四个不同的数据库中对文献进行了广泛的搜索(PubMed，Embase,Scopus,和WebofScience)。该搜索于2022年12月进行，并于2023年7月进行了更新，诸如“造血干细胞移植，骨髓移植,“\”死亡率，机会性感染，根据MeSH术语寻找与“严重联合免疫缺陷”相关的“”和“生存率”。文章的语言是\"英语,“并且仅选择了从2000年开始发表的文章。23篇文章符合审查和数据提取的纳入标准。收集的数据证实了早期的HSCT，但最重要的是,无活动性感染患者的HSCT，与更好的总体生存率有关。SCID新生儿筛查的普遍实施将是使大多数移植能够在这种“理想情况”下进行的基本支柱，并且可以避免感染。与HLA相同的同胞供者的HSCT也与更好的生存率相关，但这是最不常见的情况。出于这个原因,使用匹配的无关供体（MUD）和不匹配的相关供体（mMRD/单倍体）进行移植可作为替代方案。使用MUD获得的结果正在改善，并且显示出与MSD相似的存活率，以及他们不需要使用昂贵的技术来操纵移植物。然而,HSCT术后并发症发生率仍然很高。仅在几个大型中心进行mMRD/Haplo的移植，因为该技术在体外进行CD3/CD19消耗和TCRαβ/CD19消耗或CD34选择技术的成本很高。使用移植后环磷酰胺进行体内T细胞消耗的新可能性也可能是在没有这种技术的中心进行mMRD移植的可行替代方法。尤其是在发展中国家。
