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我们能否在婴儿期识别 WHIM?利用公共新生儿筛查过程的机会。

Can we identify WHIM in infancy? Opportunities with the public newborn screening process.

机构信息

Division of Allergy and Immunology, Department of Pediatrics and Medicine, Morsani College of Medicine, University of South Florida, Tampa, Florida, USA.

Division of Allergy and Immunology, Department of Pediatrics, Johns Hopkins All Children's Hospital, St Petersburg, Florida, USA.

出版信息

Am J Med Genet C Semin Med Genet. 2022 Jun;190(2):215-221. doi: 10.1002/ajmg.c.32002. Epub 2022 Oct 9.

Abstract

Newborn screening (NBS) for severe combined immunodeficiency (SCID) utilizing T-cell receptor excision circles (TRECs) has been implemented in all 50 states as of December 2018 and has been transformative for the clinical care of SCID patients. Though having high sensitivity for SCID, NBS-SCID has low specificity, therefore is able to detect other causes of lymphopenia in newborns including many inborn errors of immunity (IEIs). In a recent study, three of six newborns later diagnosed with Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) syndrome were found to have a low TRECs and lymphopenia at birth. This presents an opportunity to increase the detection and diagnosis of WHIM syndrome by NBS-SCID with immunological follow-up along with a combination of flow cytometry for immune cell subsets, absolute neutrophil count, and genetic testing, extending beyond the conventional bone marrow studies. Coupled with emerging technologies such as next-generation sequencing, transcriptomics and proteomics, dried blood spots used in NBS-SCID will promote earlier detection, diagnosis, and therefore treatment of IEIs such as WHIM syndrome.

摘要

截至 2018 年 12 月,利用 T 细胞受体切除环(TRECs)对严重联合免疫缺陷(SCID)进行新生儿筛查(NBS)已在全美 50 个州实施,这对 SCID 患者的临床护理产生了变革性的影响。尽管 NBS-SCID 对 SCID 具有很高的灵敏度,但特异性较低,因此能够检测到新生儿中其他导致淋巴细胞减少的原因,包括许多先天性免疫缺陷(IEI)。在最近的一项研究中,6 名后来被诊断为疣、低丙种球蛋白血症、感染和骨髓细胞缺失(WHIM)综合征的新生儿中有 3 名在出生时就存在 TRECs 低和淋巴细胞减少。这为通过 NBS-SCID 进行免疫随访,以及结合流式细胞术检测免疫细胞亚群、绝对中性粒细胞计数和基因检测,来提高 WHIM 综合征的检测和诊断提供了机会,这超出了传统的骨髓研究。与下一代测序、转录组学和蛋白质组学等新兴技术相结合,NBS-SCID 中使用的干血斑将促进对 WHIM 综合征等 IEI 的早期检测、诊断和治疗。

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