佩利兹-梅茨巴赫病神经干细胞移植后的长期安全性、免疫反应和影像学结果。
Long-Term Safety, Immunologic Response, and Imaging Outcomes following Neural Stem Cell Transplantation for Pelizaeus-Merzbacher Disease.
机构信息
Department of Neurological Surgery, University of California San Francisco, 550 16(th) Street, 4(th) Floor, San Francisco, CA 94143-0137, USA; Department of Pediatrics, University of California San Francisco, San Francisco, CA 94143, USA.
Department of Neurology, University of California San Francisco, San Francisco, CA 94143, USA; Department of Radiology and Biomedical Imaging, University of California San Francisco, San Francisco, CA 94143, USA; Bioengineering Graduate Group, University of California San Francisco & Berkeley, San Francisco, CA 94143, USA.
出版信息
Stem Cell Reports. 2019 Aug 13;13(2):254-261. doi: 10.1016/j.stemcr.2019.07.002. Epub 2019 Aug 1.
Four boys with Pelizaeus-Merzbacher disease, an X-linked leukodystrophy, underwent transplantation with human allogeneic central nervous system stem cells (HuCNS-SC). Subsequently, all subjects were followed for an additional 4 years in this separate follow-up study to evaluate safety, neurologic function, magnetic resonance imaging (MRI) data, and immunologic response. The neurosurgical procedure, immunosuppression, and HuCNS-SC transplantation were well tolerated and all four subjects were alive at the conclusion of the study period. At year 2, all subjects exhibited diffusion MRI changes at the implantation sites as well as in more distant brain regions. There were persistent, increased signal changes in the three patients who were studied up to year 5. Two of four subjects developed donor-specific HLA alloantibodies, demonstrating that neural stem cells can elicit an immune response when injected into the CNS, and suggesting the importance of monitoring immunologic parameters and identifying markers of engraftment in future studies.
四位患有 Pelizaeus-Merzbacher 病（一种 X 连锁脑白质营养不良）的男孩接受了异体人中枢神经系统干细胞（HuCNS-SC）移植。随后，在这项单独的随访研究中，所有受试者又额外随访了 4 年，以评估安全性、神经功能、磁共振成像（MRI）数据和免疫反应。神经外科手术、免疫抑制和 HuCNS-SC 移植均耐受良好，研究结束时所有 4 名受试者均存活。在第 2 年，所有受试者在植入部位和更远的脑区均出现弥散 MRI 改变。在研究至第 5 年的 3 名患者中，持续性、信号增强的改变一直存在。4 名受试者中有 2 名产生了供体特异性 HLA 同种异体抗体，表明当神经干细胞被注入中枢神经系统时会引发免疫反应，这表明在未来的研究中监测免疫参数和识别植入标记物非常重要。
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