Published online Oct 26, 2019. doi: 10.4252/wjsc.v11.i10.891
Peer-review started: March 15, 2019
First decision: June 3, 2019
Revised: June 12, 2019
Accepted: August 20, 2019
Article in press: August 20, 2019
Published online: October 26, 2019
Perinatal 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.
The aim is to evaluate the efficacy and safety of stem cell treatment in children with CP using a systematic review and meta-analysis
We 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.
We 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.
Stem 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.
Core tip: Cerebral palsy is a severe motor disability resulting from perinatal complications. Recently, stem cell therapy emerged as a promising treatment option and raised hope in patients and their families. However, high-quality randomized clinical trials investigating the efficacy of stem cell therapy as a treatment for cerebral palsy are scarce. We included the small number of currently available trials in our meta-analysis. A slight but significantly positive effect on the gross motor function favoring the stem cell group can be seen.