Abstract
The aim of this study was to evaluate the safety and efficacy of autologous bone marrow mononuclear cell transplantation combined with educational intervention for children with autism spectrum disorder. An open‐label clinical trial was performed from July 2017 to August 2019 at Vinmec International Hospital, Hanoi, Vietnam. Thirty children who fulfilled the autism criteria of the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, and had Childhood Autism Rating Scale (CARS) scores >37 were selected. Bone marrow was harvested by anterior iliac crest puncture under general anesthesia. The volume collected was as follows: 8 mL/kg for patients under 10 kg (80 mL + [body weight in kg − 10] × 7 mL) for patients above 10 kg. Mononuclear cells were isolated with a Ficoll gradient and then infused intrathecally. The same procedure was repeated 6 months later. After the first transplantation, all patients underwent 8 weeks of educational intervention based on the Early Start Denver Model. There were no severe adverse events associated with transplantation. The severity of autism spectrum disorder (ASD) was significantly reduced, with the median CARS score decreasing from 50 (range 40‐55.5) to 46.5 (range 33.5‐53.5) (P < .05). Adaptive capacity increased, with the median Vineland Adaptive Behavior Scales score rising from 53.5 to 60.5. Social communication, language, and daily skills improved markedly within 18 months after transplantation. Conversely, repetitive behaviors and hyperactivity decreased remarkably. Autologous bone marrow mononuclear cell transplantation in combination with behavioral intervention was safe and well tolerated in children with ASD (Trial registration: ClinicalTrials.gov identifier: NCT03225651).
Highlights
Autism spectrum disorder (ASD) is a complex spectrum of disorders characterized by two typical abnormalities: (a) deficits of social communication and interaction; (b) the presence of restricted interests as well as repetitive and stereotypic verbal and nonverbal behaviors.[1,2]
A combined culture-expanded/nonexpanded cell therapy study reported on the outcomes of allogenic cord blood mononuclear cell (CBMNC) transplantation vs CBMNC combined with umbilical cord mesenchymal stem cells (MSCs) transplantation for children with ASD.[29]
All participants still belonged to severe level at the baseline after receiving behavioral intervention with a mean duration of 3.5 years, this study showed improvements in various aspects after bone marrow mononuclear cell (BMMNC) transplantation combined with educational intervention
Summary
Autism spectrum disorder (ASD) is a complex spectrum of disorders characterized by two typical abnormalities: (a) deficits of social communication and interaction; (b) the presence of restricted interests as well as repetitive and stereotypic verbal and nonverbal behaviors.[1,2] Comorbidities including sleep disorders, seizures, and gastrointestinal difficulties are very common in children with ASD.[3]. Educational and behavioral interventions have been recognized as crucial for the management of ASD in children.[12] The evidence indicates that young children with ASD benefit from interventions that focus on improving social interaction, communication, and challenging behaviors.[13,14] many children who receive those treatments remain significantly impaired.[15]. In BTBR mice, Segal-Gavish et al showed that transplantation of MSCs resulted in a reduction in stereotypic behaviors, a decrease in cognitive rigidity, and an improvement in social behavior. It has been shown that brain-derived neurotrophic factor protein levels as well as neurogenesis increased in the hippocampus following stem cell treatment.[23] Perets et al demonstrated that brainderived neurotrophic factors secreted by transplanted MSCs were a key factor in the observed reductions in stereotypic behavior and in the improvement of cognitive flexibility in the BTBR model.[24] Ha et al revealed that transplanted human adipose-derived stem cells. The combination of cell therapy and educational intervention may improve clinical manifestations such as social communication, language, and daily skills in children with ASD
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