Effects of Cerebrolysin on the Neurodevelopmental Outcomes in Infants with Down Syndrome: A Randomized Controlled Pilot Trial

ABSTRACT Introduction Down syndrome (DS) is a common cause of intellectual disability with a possible glial-neuronal disruption. We assessed the effects of Cerebrolysin on the neurodevelopmental outcomes of infants with DS. Methods This randomized controlled pilot trial included 64 infants with DS. They were allocated into a treatment group (n =32) who received weekly intramuscular Cerebrolysin injections for 12 months and a control group (n =32) who did not receive Cerebrolysin. We assessed the five domains of neurodevelopment (expressive communication, receptive communication, fine motor, gross motor, and cognitive development) for both groups at the ages of 6 months (basal visit), 12 months (first follow-up visit) and 18 months (second follow-up visit), using Bayley Scales of infant and toddler development (third edition). The secondary outcome was to detect the occurrence of Cerebrolysin-related side effects among the treatment group. Kendall’s tau-b correlation coefficient, chi-square test, Fisher’s exact test, Student's t-test, Mann-Whitney and Wilcoxon signed-rank tests were used for the statistical analysis. Results Infants of both groups were matched with respect to gender, maternal level of education, their initial growth parameters, nutritional status, and socioeconomic status. No statistically significant differences existed between both groups regarding all neurodevelopmental domains on the basal visit, while on subsequent visits Z-scores improved in the treatment group, but not in the controls. Within the treatment group, there was a significant improvement in all domains at 12 and 18 months of age (p < .001). The improvement was mainly during the first 6 months of the study (p < .001 for all domains), and was best for fine motor and cognitive development. Only one infant in the treatment group developed fits and was ruled out from the study. Conclusion Weekly intramuscular injections of Cerebrolysin during the first year of life could possibly improve the neurodevelopmental outcomes and mental health in infants with DS. Further, large-scale studies are still needed to evaluate the efficacy and safety of Cerebrolysin among this sector of disabled infants.