Safety and Efficacy of Cerebrolysin in Infants with Communication Defects due to Severe Perinatal Brain Insult: A Randomized Controlled Clinical Trial

BACKGROUND AND PURPOSE: The neuroregenerative drug Cerebrolysin has demonstrated efficacy in improving cognition in adults with stroke and Alzheimer's disease. The aim of this study was to determine the efficacy and safety of Cerebrolysin in the treatment of communication defects in infants with severe perinatal brain insult. METHODS: A randomized placebo-controlled clinical trial was conducted in which 158 infants (age 6-21 months) with communication defects due to severe perinatal brain insult were enrolled; 120 infants completed the study. The Cerebrolysin group (n=60) received twice-weekly Cerebrolysin injections of 0.1 mL/kg body weight for 5 weeks (total of ten injections). The placebo group (n=60) received the same amount and number of normal saline injections. RESULTS: The baseline Communication and Symbolic-Behavior-Scale-Developmental Profile scores were comparable between the two groups. After 3 months, the placebo group exhibited improvements in the social (p<0.01) and speech composite (p=0.02) scores, with 10% and 1.5% increases from baseline, respectively. The scores of the Cerebrolysin group changed from concern to no concern, with increases of 65.44%, 45.54%, 358.06%, and 96.00% from baseline in the social (p<0.001), speech (p<0.001), symbolic (p<0.001), and total (p<0.001) scores. CONCLUSIONS: Cerebrolysin dramatically improved infants' communication especially symbolic behavior which positively affected social interaction. These findings suggest that cerebrolysin may be an effective and feasible way equivalent to stem cell therapy.