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首页> 美国卫生研究院文献>other >Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Stem Cell Transplantation (HCT) for Severe Combined Immunodeficiency (SCID) Patients: a Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT
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Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Stem Cell Transplantation (HCT) for Severe Combined Immunodeficiency (SCID) Patients: a Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT

机译：严重联合免疫缺陷病（SCID）患者在造血干细胞移植（HCT）后晚期效应方面的最新知识和未来研究的优先权：第二届小儿血液和骨髓移植联合会国际会议关于小儿HCT晚期效应的共识声明

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Severe Combined Immunodeficiency (SCID) is one of the most common indications for pediatric hematopoietic cell transplantation (HCT) in patients with primary immunodeficiency (PID). Historically, SCID was diagnosed in infants who presented with opportunistic infections within the first year of life. With newborn screening (NBS) for SCID in most of the U.S., the majority of infants with SCID are now diagnosed and treated in the first 3.5 months of life, although in the rest of the world, the lack of NBS means that most infants with SCID still present with infections. The average survival for transplanted SCID patients currently is >70% at 3 years post-transplant, although this can vary significantly based on multiple factors including age and infection status at the time of transplantation, type of donor source utilized, manipulation of graft prior to transplant, GVHD prophylaxis, type of conditioning (if any) utilized and underlying genotype of SCID. In at least one study of SCID patients who received no conditioning, long-term survival was 77% at 8.7 years (range out to 26 years) post-transplantation. While a majority of patients with SCID will engraft T cells without any conditioning therapy, depending on genotype, donor source, HLA match and presence of circulating maternal cells a sizable percentage of these will fail to achieve full immune reconstitution. Without conditioning, T cell reconstitution typically occurs, although not always fully, while B cell engraftment does not—leaving some molecular types of SCID patients with intrinsically defective B cells in most cases dependent on regular infusions of immunoglobulin. Because of this, many centers have used conditioning with alkylating agents including busulfan or melphalan known to open marrow niches in attempts to achieve B cell reconstitution. Thus, it is imperative that we understand the potential late effects of these agents in this patient population. There are also non-immunologic risks associated with HCT for SCID that appear to be dependent upon the genotype of the patient. In this report we have evaluated the published data on late effects and attempted to summarize the known risks associated with conditioning and alternative donor sources. These data, while informative, are also a clear demonstration that there is still much to be learned from the SCID population in terms of their post-HCT outcomes. This paper will summarize current findings and recommend further research in areas considered high priority. Specific guidelines regarding a recommended approach to long-term follow up, including laboratory and clinical monitoring will be forthcoming in a subsequent paper.
机译：严重合并免疫缺陷（SCID）是原发性免疫缺陷（PID）患者小儿造血细胞移植（HCT）的最常见适应症之一。从历史上看，SCID被诊断为出生后第一年出现机会性感染的婴儿。在美国大部分地区，通过对SCID进行新生儿筛查（NBS），大多数SCID婴儿现在已经在出生后的3.5个月内得到了诊断和治疗，尽管在世界其他地区，缺乏NBS意味着大多数患有SCID的婴儿SCID仍然存在感染。目前，移植的SCID患者在移植后3年的平均存活率> 70％，尽管这可能会因多种因素而发生显着变化，这些因素包括移植时的年龄和感染状况，使用的供体来源类型，移植前的移植物操作移植，GVHD预防，利用的调节类型（如有）和SCID的潜在基因型。在至少一项没有接受任何调节的SCID患者的研究中，移植后8.7年（范围为26年）的长期存活率为77％。尽管大多数SCID患者无需进行任何条件治疗即可移植T细胞，但取决于基因型，供体来源，HLA匹配情况和循环中的母体细胞的存在，其中相当一部分无法实现完全的免疫重建。如果不进行调节，通常会发生T细胞重构，尽管并不总是完全发生，而B细胞移植却没有，因此在某些情况下，SCID患者的某些分子类型具有固有缺陷的B细胞，在大多数情况下取决于定期输注免疫球蛋白。因此，许多中心都在使用烷化剂（包括白消安或美法仑）进行调理，这些试剂已知会打开骨髓壁m，以实现B细胞的重建。因此，我们必须了解这些药物在该患者人群中的潜在晚期作用。还存在与SCID的HCT相关的非免疫风险，这似乎取决于患者的基因型。在本报告中，我们评估了已发布的有关后期影响的数据，并试图总结与调节和替代供体来源相关的已知风险。这些数据虽然提供了信息，但也清楚地表明，从SCID人群的HCT后结果来看，仍有很多值得学习的地方。本文将总结当前的发现，并建议在高度优先的领域中进行进一步的研究。有关推荐的长期随访方法的具体指南，包括实验室和临床监测，将在随后的论文中发表。

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