Abstract

BACKGROUNDPerinatal complications may result in life-long morbidities, among which cerebral palsy (CP) is the most severe motor disability. Once developed, CP is a non-progressive disease with a prevalence of 1-2 per 1000 live births in developed countries. It demands an extensive and multidisciplinary care. Therefore, it is a challenge for our health system and a burden for patients and their families. Recently, stem cell therapy emerged as a promising treatment option and raised hope in patients and their families.AIMThe aim is to evaluate the efficacy and safety of stem cell treatment in children with CP using a systematic review and meta-analysisMETHODSWe performed a systematic literature search on PubMed and EMBASE to find randomized controlled clinical trials (RCT) investigating the effect of stem cell transplantation in children with CP. After the review, we performed a random-effects meta-analysis focusing on the change in gross motor function, which was quantified using the gross motor function measure. We calculated the pooled standardized mean differences of the 6- and/or 12-mo-outcome by the method of Cohen. We quantified the heterogeneity using the I-squared measure.RESULTSWe identified a total of 8 RCT for a qualitative review. From the initially selected trials, 5 met the criteria and were included in the meta-analysis. Patients’ population ranged from 0.5 up to 35 years (n = 282). We detected a significant improvement in the gross motor function with a pooled standard mean difference of 0.95 (95% confidence interval: 0.13-1.76) favoring the stem cell group and a high heterogeneity (I2 = 90.1%). Serious adverse events were rare and equally distributed among both intervention and control groups.CONCLUSIONStem cell therapy for CP compared with symptomatic standard care only, shows a significant positive effect on the gross motor function, although the magnitude of the improvement is limited. Short-term safety is present and further high-quality RCTs are needed.

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