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Clinical Trial
. 2025 May 30;16(1):268.
doi: 10.1186/s13287-025-04404-4.

Outcomes of autologous bone marrow mononuclear cell administration combined with educational intervention in the treatment of autism spectrum disorder: a randomized, open-label, controlled phase II clinical trial

Affiliations
Clinical Trial

Outcomes of autologous bone marrow mononuclear cell administration combined with educational intervention in the treatment of autism spectrum disorder: a randomized, open-label, controlled phase II clinical trial

Liem Thanh Nguyen et al. Stem Cell Res Ther. .

Abstract

Background: 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).

Methods: 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.

Results: 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).

Conclusions: Intrathecal BMMNC combined with an educational intervention improved disease severity and adaptability more than education alone in children with ASD.

Trial registration: clinicaltrials.gov, NCT05307536. Date registered 12 February 2022. http://clinicaltrials.gov/study/NCT05307536 .

Keywords: Autism spectrum disorder; Bone marrow mononuclear cells; Cell therapy; Intrathecal infusion; Randomized phase II clinical trial.

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Conflict of interest statement

Declarations. Ethics approval and consent to participate: The study was approved by the National Ethics Committee of the Vietnam Ministry of Health, with approval number 120/CN-HDDD. Title of the approved project: “Autologous Bone Marrow Mononuclear Cell Transplantation Combined with Educational Intervention for Autism Spectrum Disorders (ASD): A Phase II Randomized Controlled Trial”; Name of the institutional approval committee: The National Ethics Committee of the Vietnam Ministry of Health (Hanoi, Vietnam); Approval number: 120/CN-HDDD; Date of approval: November 05, 2021. The patients’ guardians or legally authorized representatives provided written informed consent for their participation in the study and the use of samples. The consent explicitly included permission to use patient identifiers, such as age and sex, for publication purposes. All treatments, including clinical examinations, laboratory tests, brain MRI, EEG, genetic tests, cell collection, cell processing, cell administration, hospital stay fees, and educational interventions, were provided free of charge to the participants and their families. Consent for publication: Not applicable. Competing interests: The authors declare that they have no competing interests.

Figures

Fig. 1
Fig. 1
CONSORT diagram of the study. This flow diagram illustrates the patient enrollment process, randomization, allocation, follow-up, and analysis in the study. A total of 87 patients were screened, resulting in 54 patients who were randomized into two groups. The CT group (n = 27) received stem cell therapy (SCT) in combination with an educational intervention. During follow-up, one patient discontinued on day 30, and another discontinued on day 60. The control group received only the educational intervention. During follow-up, one patient discontinued on day 80, and another discontinued on day 180. A total of 25 patients in each group completed the study through day 365 and were included in the final analysis
Fig. 2
Fig. 2
The CARS score decreased significantly more in the CT group than in the control group. This figure shows the trajectories of the CARS scores for individual participants in the CT group and the control group over four time points: baseline, two, six months, and 12 months. Each line represents an individual participant’s CARS score over time. The shaded areas represent the mean trend and variability within each group. The CARS score decreased significantly more in the CT group than in the control group, indicating greater improvement in autism-related symptoms for participants in the CT group

References

    1. Simonoff E, Pickles A, Charman T, Chandler S, Loucas T, Baird G. Psychiatric disorders in children with autism spectrum disorders: prevalence, comorbidity, and associated factors in a population-derived sample. J Am Acad Child Adolesc Psychiatry. 2008;47(8):921–9. - PubMed
    1. Baio J. Prevalence of autism spectrum disorder among children aged 8 years—autism and developmental disabilities monitoring network, 11 sites, united States, 2014. MMWR Surveillance Summaries. 2018;67. - PMC - PubMed
    1. Kousha M, Attar HA, Shoar Z. Anxiety, depression, and quality of life in Iranian mothers of children with autism spectrum disorder. J Child Health Care. 2016;20(3):405–14. - PubMed
    1. Leigh JP, Du J. Brief report: forecasting the economic burden of autism in 2015 and 2025 in the united States. J Autism Dev Disord. 2015;45:4135–9. - PubMed
    1. Enstrom AM, Van de Water JA, Ashwood P. Autoimmunity in autism. Curr Opin Invest Drugs (London England: 2000). 2009;10(5):463. - PMC - PubMed

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