Nguyen Liem Thanh, Nguyen Phuong Mai, Nguyen Hoang-Phuong, Bui Hau Thi, Dao Lan Thi Mai, Van Pham Minh, Hoang Chi Khanh, Nguyen Phuong Thi, Nguyen Thao Thi Phuong, Nguyen Anh Thi Phuong, Hoang Van Thi, Bui Hoa Thi Phuong, Vuong Ngan Kim, Van Ngo Doan
Vinmec Research Institute of Stem Cell and Gene Technology (VRISG), College of Health Sciences, VinUniversity, Vinhomes Ocean Park, Gia Lam, Hanoi, 100000, Vietnam.
Vinmec Times City International Hospital, Vinmec Health Care System, 458 Minh Khai Street, Hai Ba Trung, Hanoi, 100000, Vietnam.
Stem Cell Res Ther. 2025 May 30;16(1):268. doi: 10.1186/s13287-025-04404-4.
This study evaluated the effectiveness of intrathecal autologous bone marrow mononuclear cell (BMMNC) therapy combined with education compared with education alone for the treatment of autism spectrum disorder (ASD).
Fifty-four children with ASD, aged three to seven years, were randomly assigned to two groups. Fifty patients completed the study (25 patients per group). The cell therapy (CT) group received two BMMNC infusions six months apart along with an educational intervention, while the control group received education only. Efficacy outcomes were assessed at baseline, two, six, and 12 months, based on: (1) changes in ASD severity evaluated through the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5), the Childhood Autism Rating Scale (CARS), and the Clinical Global Impression-Severity (CGI-S) scale scores and (2) improvements in social interaction, adaptive behavior, and daily living skills measured by the Vineland Adaptive Behavior Scales (VABS-II) and Clinical Global Impression-Improvement (CGI-I) scale scores.
At 12 months, the CT group presented a 48.0% reduction in individuals classified at the most severe DSM-5 level compared with 8.0% in the control group (p = 0.004). The CARS scores were significantly lower in the CT group (-5.9 points) than in the control group (-1.5 points) (p < 0.0001). Similarly, the CT group exhibited greater improvement in CGI-S scores (-1.5 points) than did the control group (-0.1 points) (p < 0.0001). The VABS-II scores increased by 8.5 points in the CT group versus 1.4 points in the control group (p < 0.0001). Finally, the CGI-I scores improved from 2.8 to 2.0 in the CT group but worsened from 3.0 to 3.5 in the control group (p < 0.0001).
Intrathecal BMMNC combined with an educational intervention improved disease severity and adaptability more than education alone in children with ASD.
clinicaltrials.gov, NCT05307536. Date registered 12 February 2022. http://clinicaltrials.gov/study/NCT05307536 .
本研究评估了鞘内注射自体骨髓单个核细胞（BMMNC）疗法联合教育与单纯教育相比，对治疗自闭症谱系障碍（ASD）的有效性。
54名年龄在3至7岁的ASD儿童被随机分为两组。50名患者完成了研究（每组25名患者）。细胞治疗（CT）组每隔6个月接受两次BMMNC输注，并接受教育干预，而对照组仅接受教育。在基线、2个月、6个月和12个月时评估疗效结果，评估依据为：（1）通过《精神疾病诊断与统计手册》第五版（DSM-5）、儿童自闭症评定量表（CARS）和临床总体印象-严重程度（CGI-S）量表评分评估的ASD严重程度变化；（2）通过文兰适应行为量表（VABS-II）和临床总体印象-改善（CGI-I）量表评分衡量的社交互动、适应性行为和日常生活技能的改善情况。
在12个月时，CT组中被归类为DSM-5最严重级别的个体减少了48.0%，而对照组为8.0%（p = 0.004）。CT组的CARS评分（-5.9分）显著低于对照组（-1.5分）（p < 0.0001）。同样，CT组在CGI-S评分方面的改善（-1.5分）大于对照组（-0.1分）（p < 0.0001）。CT组的VABS-II评分增加了8.5分，而对照组增加了1.4分（p < 0.0001）。最后，CT组的CGI-I评分从2.8改善到2.0，而对照组从3.0恶化到3.5（p < 0.0001）。
对于患有ASD的儿童，鞘内注射BMMNC联合教育干预比单纯教育更能改善疾病严重程度和适应性。
clinicaltrials.gov，NCT05307536。注册日期为2022年2月12日。http://clinicaltrials.gov/study/NCT05307536 。
