人神经前体细胞移植治疗儿童广泛性发育障碍初探

Transplantation of human neural precursor cells in the treatment of children with pervasive developmental disorder

摘要:

目的：观察人神经前体细胞（hNPCs）移植治疗儿童广泛性发育障碍的安全性及疗效。方法：对22例广泛性发育障碍患儿进行研究，其中孤独症9例，Rett综合征13例，经颅骨钻孔侧脑室穿刺注射hNPCs治疗。分别于手术前、术后1个月、术后6个月、术后1年采用孤独症行为评定量表（ABC）对移植患儿进行评估。结果：治疗及随访过程中无严重不良事件发生。移植后22例患儿中，1例失访，17例患儿临床症状出现不同程度的改善，其中孤独症患儿8例，Rett综合征患儿9例。孤独症患儿行ABC量表评估后发现，术后6个月交往因子得分，以及术后1年ABC总分、交往及语言因子得分均低于术前，差异有统计学意义（P<0.05）。结论：临床应用hNPCs移植治疗儿童广泛性发育障碍是安全、可行的，并具有一定疗效，有必要扩大观察病例数进一步研究。

Abstract:

OBJECTIVE: To assess the efficiency and safety of human neural progenitor cells (hNPCs) transplantation in the treatment of pervasive developmental disorder (PDD) in children. METHODS: Twenty-two children with PDD were treated, including 13 children with Rett syndrome and 9 children with autism. They accepted hNPCs transplantation voluntarily. hNPCs derived from aborted fetal tissue were injected into the lateral ventricle of the patients under supersonic guidance. All patients were assessed according to the Autism Behavior Checklist before operation, at one and six months post operation, and one year later. RESULTS: No delayed complications resulting from this therapy were observed. The clinical symptoms of 17 patients, including 8 patients with autism and 9 patients with Rett syndrome, improved in varying degrees. The assessment results of the Autism Behavior Checklist for children with autism showed that compared with pre-operative function, social communication scores were significantly reduced at six months after transplantation, and total scores and social communication and language scores were also significantly reduced 1 year after transplantation (P<0.05). CONCLUSIONS: These results suggest that hNPCs transplantation is effective and safe for treatment of PPD in children. It deserves a further study