Cell Transplant. 2013;22 Suppl 1:S101-12. doi: 10.3727/096368913X672163. Epub 2013 Sep 10.
The purpose of this study was to investigate the clinical efficacy of neural stem/progenitor cell (NS/PC) transplantation to treat severe cortical visual impairment (CVI), a sequela of neonatal brain injury. Fifty-two patients with cerebral injury and CVI were randomly divided into two groups: the treatment group (n = 25, with the median age of 18 months) and the control group (n = 27, with the median age of 19.5 months). The treatment group received intracerebroventricular transplantation of human NS/PCs and rehabilitation training. The control group received rehabilitation only. The visual function was assessed by Holt's method at various time points after transplantation. One in five patients with fundus abnormalities accompanied by blindness regained light perception. The visual functions of 75% of the patients with normal fundus were improved by one level or more in a 2-year follow-up. The median efficacy appeared 60 days posttransplantation. The total effective rate of cell transplantation on visual improvement was 64% (16 patients of 25), among which one blind patient regained light perception, five (31.2%) CVI patients improved by one level, and 10 (62.5%) improved by more than one level. Functional magnetic resonance imaging (fMRI) in a subpopulation of patients showed enhanced signals in the occipital lobe, visual pathway, and apical lobe after transplantation. In the control group, four patients with fundus abnormalities showed no improvement. Nine of 23 CVI patients with normal fundus improved visual function by more than one level. At the 2-year follow-up, no blind patients showed visual improvement. The total effective rate was 33.33% (9 of 27 patients). Among those showing visual improvement in the control group, six patients (66.67%) improved by one level, and three (33.33%) by more than one level. The median efficacy occurred in 365 days. Human NS/PC transplantation is effective to treat patients with severe CVI after neonatal brain injury. Compared with the traditional rehabilitation training, cell transplantation showed not only earlier visual improvement but also higher improvement rates and degrees. This article is published as part of the International Association of Neurorestoratology (IANR) supplement issue of Cell Transplantation.
本研究旨在探讨神经干细胞/祖细胞（NS/PC）移植治疗新生儿脑损伤后严重皮质视觉障碍（CVI）的临床疗效。52 例脑损伤合并 CVI 的患者被随机分为两组：治疗组（n=25，中位年龄 18 个月）和对照组（n=27，中位年龄 19.5 个月）。治疗组接受脑室注射人 NS/PC 并进行康复训练，对照组仅进行康复训练。移植后不同时间点采用霍尔特法评估视觉功能。眼底异常伴失明的患者中有五分之一恢复光感。在 2 年随访中，75%眼底正常的患者视觉功能提高 1 级或以上。移植后 60 天出现中位疗效。细胞移植对视觉改善的总有效率为 64%（25 例中有 16 例），其中 1 例盲患者恢复光感，5 例（31.2%）CVI 患者提高 1 级，10 例（62.5%）提高 1 级以上。对部分患者进行功能磁共振成像（fMRI）检查显示，移植后枕叶、视觉通路和顶叶信号增强。对照组 4 例眼底异常患者无改善，23 例眼底正常的 CVI 患者中有 9 例视觉功能提高 1 级以上。2 年随访时，无盲患者出现视觉改善，总有效率为 33.33%（27 例中有 9 例）。对照组中，6 例（66.67%）提高 1 级，3 例（33.33%）提高 1 级以上。中位疗效发生在 365 天。人 NS/PC 移植治疗新生儿脑损伤后严重 CVI 有效。与传统康复训练相比，细胞移植不仅能更早地改善视力，而且提高程度和提高率更高。本文作为国际神经修复学会（IANR）细胞移植专刊的一部分发表。
