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早期接种卡介苗对采用低强度预处理方案的小儿严重联合免疫缺陷病患者造血干细胞移植结局的影响。

Effect of Early Bacillus Calmette-Guerin Vaccination of Pediatric Severe Combined Immunodeficiency Patients on the Outcome of Hematopoietic Stem Cell Transplantation Using a Reduced-Intensity Conditioning Regimen.

机构信息

Pediatric Cell and Gene Therapy Research Center, Gene, Cell & Tissue Research Institute, Tehran University of Medical Science, Tehran, Iran.

Pediatric Cell and Gene Therapy Research Center, Gene, Cell & Tissue Research Institute, Tehran University of Medical Science, Tehran, Iran.

出版信息

Transplant Cell Ther. 2023 Mar;29(3):188.e1-188.e8. doi: 10.1016/j.jtct.2022.12.007. Epub 2022 Dec 17.

Abstract

The eminence of Bacillus Calmette-Guerin (BCG) vaccine in newborn vaccination programs has been conspicuous throughout the years, especially in low-income developing countries where tuberculosis is prevalent; however, application of the BCG vaccine is not without constraints, especially in patients afflicted with immunodeficiency diseases, such as severe combined immunodeficiency (SCID). The present study aimed to evaluate whether the administration of BCG vaccine at birth could improve the outcomes of hematopoietic stem cell transplantation (HSCT) in pediatric patients with SCID. In this study, 30 SCID patients who underwent HSCT using a reduced-intensity conditioning regimen (RIC) were followed-up for 2 years post-HSCT. The outcomes of HSCT were evaluated in both non-BCG-vaccinated patients (n = 12) and BCG-vaccinated patients (n = 18). Our results show a higher incidence of acute graft-versus-host disease (aGVHD), but not of chronic GVHD, in the BCG-vaccinated patients, and a similar overall survival (OS) rate in the 2 groups. We speculate that the similar OS rate in the 2 groups, despite the risk of BGC vaccination, was because this group received an RIC conditioning regimen. There was no other difference between the 2 groups. Considering the effect of the BCG vaccine on HSCT outcome, we suggest that the administration of BCG vaccine be deferred until age 3 months so that APT testing without the interference of maternal antibodies can be performed. However, this study could benefit from a larger cohort to further validate our findings, as the possible reason for some factors not being statistically significant was our small sample size.

摘要

卡介苗(BCG)疫苗在新生儿疫苗接种计划中的卓越地位多年来一直引人注目,尤其是在结核病流行的低收入发展中国家;然而,BCG 疫苗的应用并非没有限制,特别是在患有免疫缺陷疾病的患者中,例如严重联合免疫缺陷(SCID)。本研究旨在评估在患有 SCID 的儿科患者中,出生时接种 BCG 疫苗是否可以改善造血干细胞移植(HSCT)的结果。在这项研究中,对 30 名接受低强度预处理方案(RIC)HSCT 的 SCID 患者进行了 2 年的随访。在未接种 BCG 疫苗的患者(n=12)和接种 BCG 疫苗的患者(n=18)中评估了 HSCT 的结果。我们的结果显示,接种 BCG 疫苗的患者发生急性移植物抗宿主病(aGVHD)的发生率较高,但慢性 GVHD 的发生率较低,两组的总生存率(OS)相似。我们推测,尽管存在 BGC 接种的风险,但两组的 OS 率相似,是因为该组接受了 RIC 预处理方案。两组之间没有其他差异。考虑到 BCG 疫苗对 HSCT 结果的影响,我们建议将 BCG 疫苗的接种推迟到 3 个月大,以便在没有母体抗体干扰的情况下进行 APT 检测。然而,由于我们的样本量较小,一些因素没有达到统计学意义,这可能是导致本研究的原因,因此这项研究可以从更大的队列中受益,以进一步验证我们的发现。

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