Introduction
Global developmental delay (GDD) can be defined as a clinical presentation that has a various etiologic profile and is a subset of developmental disabilities defined as significant delay in two or more of the following developmental domains; gross/fine motor, speech/language, cognition, social/personal behavior, and activities of daily living. As most GDD patients have limited improvements with conventional treatments, stem cell therapy can be an emerging therapeutic option. This study aims to determine the efficacy and safety of allogenic umbilical cord blood (UCB) therapy for children with GDD.
Materials and Methods
We conducted a prospective study including total of 13 children with GDD. Allogenic UCB units were selected from UCB bank of our organization. The conditions for UCB selection were at least 4 out of 6 matching HLA typing for A, B, and DRB1 antigens. Cyclosporine was administered orally from 3 days before to 13 days after allogenic UCB injection. After thawing and washing according to our protocol, UCB was infused intravenously. Raw scores of Bayley scales of infant development-II (BSID-II) were followed at baseline, 3, 6, and 12 months. Secondary outcomes such as gross motor function measure, (GMFM) and Wee Functional Independent Measure (WeeFIM) were also evaluated at the same period. Adverse events were recorded through the whole study period.
Results
The demographics of 13 eligible patients are described in Table 1. No adverse events were reported during 1 year follow-up. BSID-II was improved significantly at 3, 6 and 12 months after UCB injection for mental score (p=0.005, 0.003 and 0.003, respectively) and motor score (p=0.038, 0.005, and 0.005, respectively). Also GMFM revealed significant improvements at 6 and 12 months after treatment (p=0.006 and 0.009, respectively). Wee-FIM also showed improvements respectively in motor, cognitive and total score at 6 months (p=0.027, 0.041, and 0.027 for motor, cognitive and total WeeFIM, respectively) and 12 months (p=0.011, 0.015, and 0.011 for motor, cognitive and total WeeFIM, respectively) after treatment.
Conclusion
Allogenic UCB therapy is a safe and effective treatment option for children with GDD.
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