Affiliation:
1. Department of Child Development KK Women's and Children's Hospital (KKH) Singapore
2. Yong Loo Lin School of Medicine, National University of Singapore Singapore
3. Duke‐NUS Medical School Singapore
4. Lee Kong Chian School of Medicine Singapore
5. Singapore Cord Blood Bank Singapore
6. Children's Blood and Cancer Centre, KK Women's and Children's Hospital Singapore
7. Department of Obstetrics and Gynaecology KK Women's and Children's Hospital Singapore
Abstract
AbstractThis study aimed to document the safety and efficacy of a single infusion of autologous umbilical cord blood (UCB) in 20 autistic children aged 24–72 months. A pre‐post treatment within‐subjects open label design was used. At T = 0, 6, 12, and 18 months, participants underwent detailed and structured safety evaluations (via caregiver report), Vineland Adaptive Behavior Scale (Vineland‐3), Stanford Binet Intelligence Scale (SB‐5), Expressive One‐Word Picture Vocabulary Test, Brief Observation of Social Communication Change (BOSCC), Pervasive Developmental Disorder‐Behavior Inventory, Repetitive Behavior Scale‐Revised, Sensory Experience Questionnaire (SEQ‐2.1), Child Behavior Checklist, Clinical Global Impression‐Severity and Improvement (CGI‐I) Scales, and eye‐gaze tracking. UCB infusion was conducted at T = 6 months, hence, 0–6 months was the control period, and 6–18 months the follow‐up period. Of 20 children recruited, 19 completed the study and 1 was withdrawn due to UCB not meeting quality control criteria for infusion. There were 15 males and 4 females with an overall mean (SD) age of 4.15 (0.62) years. Mean (SD) cell dose administered was 38.16 (9.82) million cells/kg. None suffered serious adverse events although there were mild behavioral side effects and one unit grew coagulase negative staphylococcus from a post‐thaw sample. There were no significant differences in Vineland‐3, SB‐5, BOSCC, and SEQ‐2.1 scores at T = 12 and T = 18 months. Twelve participants had T = 18 CGI‐I scores of 2–3 (minimally to much improved), seven participants had scores of 4 (no change). Autologous UCB infusion in autistic children is generally safe but not without risks, including that of infection. In this within‐subjects study, some children showed global symptom improvements while others showed no change. Stem cell therapies for autism should only be conducted under strict clinical trial conditions with clear risk discussions.