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Outcomes of bone marrow mononuclear cell transplantation combined with interventional education for autism spectrum
Liem Nguyen Thanh1, Hoang-Phuong Nguyen1, Minh Duy Ngo2
1Vinmec Research Institute of Stem Cell and Gene Technology (VRISG), Hanoi, Vietnam.
Insights
Autologous bone marrow mononuclear cell transplantation safely improved autism spectrum disorder (ASD) symptoms in children. This treatment, combined with educational intervention, significantly reduced ASD severity and enhanced adaptive behaviors.
Area of Science:
- Regenerative Medicine
- Neurodevelopmental Disorders
- Clinical Trials
Background:
- Autism spectrum disorder (ASD) presents significant challenges in social communication and behavior.
- Existing interventions for ASD have varying degrees of efficacy, necessitating novel therapeutic approaches.
- Autologous bone marrow mononuclear cells (BM-MNCs) offer potential regenerative properties.
Purpose of the Study:
- To evaluate the safety and efficacy of autologous bone marrow mononuclear cell transplantation combined with educational intervention for children with autism spectrum disorder.
- To assess the impact of this combined therapy on ASD severity and adaptive behaviors.
Main Methods:
- An open-label clinical trial involving 30 children diagnosed with ASD.
- Autologous bone marrow was harvested, mononuclear cells isolated, and infused intrathecally twice, 6 months apart.
- Patients received 8 weeks of Early Start Denver Model educational intervention post-transplantation.
Main Results:
- No severe adverse events were reported, indicating a safe treatment profile.
- Significant reduction in autism severity, evidenced by a decrease in Childhood Autism Rating Scale (CARS) scores (P < .05).
- Marked improvements in adaptive capacity (Vineland Adaptive Behavior Scales), social communication, language, and daily skills; reduction in repetitive behaviors and hyperactivity.
Conclusions:
- Autologous bone marrow mononuclear cell transplantation combined with behavioral intervention is a safe and well-tolerated treatment for children with ASD.
- The combined approach demonstrates significant efficacy in ameliorating core ASD symptoms and improving overall adaptive functioning.
- This therapeutic strategy holds promise for improving outcomes in children with autism spectrum disorder.
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).
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