Sharifzadeh Narges, Ghasemi Ali, Tavakol Afshari Jalil, Moharari Fatemeh, Soltanifar Atefeh, Talaei Ali, Pouryousof Hamid Reza, Nahidi Mahsa, Fayyazi Bordbar Mohammad Reza, Ziaee Maliheh
Psychiatry and Behavioral Sciences Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.
Department of Pediatric Hematology and Oncology, Dr Sheikh Pediatric Hospital, Mashhad University of Medical Sciences, Mashhad, Iran.
Asia Pac Psychiatry. 2021 Jun;13(2):e12445. doi: 10.1111/appy.12445. Epub 2020 Nov 4.
This study aimed to determine the safety and efficacy of treatment with autologous bone marrow mesenchymal stem cell (BMMSCs) compared with the routine treatment in children with autism spectrum disorder (ASD).
In this ethically approved randomized controlled trial, 32 ASD children aged 5-15 years were randomly assigned to receive either autologous BMMSC plus rehabilitation therapy and risperidone (intervention group) or rehabilitation therapy and risperidone (control group). Autologous BMMSCs were intrathecally injected in the intervention group twice in 4 weeks. Patients were assessed using childhood autism rating scale (CARS), Gilliam autism rating scale-second edition (GARS-II), and clinical global impression (CGI) at the baseline, as well as 6 and 12 months after intervention.
Overall, 32 patients in two groups of intervention (n = 14) and control (n = 18) completed the study, of which 27 (84.4%) were male. Mean age was 9.50 ± 2.14 years. The improvements in CARS total score, GARS-II autism index, and CGI global improvement showed no significant differences between the groups over 12 months. However, the main effect for time*group interaction was significant regarding the CGI-severity of illness, showing a significantly more pronounced improvement in the intervention group (F = 6.719; P = .002).
Intrathecal injection of autologous BMMSCs seems to be safe and feasible, but has limited clinical efficacy in treatment of children with ASD.
本研究旨在确定与常规治疗相比，自体骨髓间充质干细胞（BMMSCs）治疗自闭症谱系障碍（ASD）儿童的安全性和有效性。
在这项经伦理批准的随机对照试验中，32名年龄在5至15岁的ASD儿童被随机分配接受自体BMMSC联合康复治疗及利培酮（干预组）或康复治疗及利培酮（对照组）。干预组在4周内分两次鞘内注射自体BMMSCs。在基线以及干预后6个月和12个月时，使用儿童自闭症评定量表（CARS）、 Gilliam自闭症评定量表第二版（GARS-II）和临床总体印象（CGI）对患者进行评估。
总体而言，干预组（n = 14）和对照组（n = 18）的32名患者完成了研究，其中27名（84.4%）为男性。平均年龄为9.50 ± 2.14岁。在12个月内，两组在CARS总分、GARS-II自闭症指数和CGI总体改善方面的改善无显著差异。然而，关于CGI疾病严重程度，时间*组交互作用的主效应显著，显示干预组有更明显的改善（F = 6.719；P = 0.002）。
鞘内注射自体BMMSCs似乎是安全可行的，但在治疗ASD儿童方面临床疗效有限。
