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Stem cell treatment and cerebral palsy: Systemic review and meta-analysis
Simone Eggenberger1, Céline Boucard2, Andreina Schoeberlein2
1Department of Obstetrics and Gynaecology, Inselspital, University Hospital Bern, Bern 3010, Switzerland.
Insights
Stem cell therapy shows a significant improvement in gross motor function for children with cerebral palsy (CP). While safe in the short term, the observed motor gains are modest, necessitating further high-quality research.
Area of Science:
- Regenerative Medicine
- Pediatric Neurology
- Clinical Trials
Background:
- Cerebral palsy (CP) is a severe motor disability resulting from perinatal complications, impacting 1-2 per 1000 live births.
- CP necessitates extensive multidisciplinary care, posing significant challenges to healthcare systems and families.
- Stem cell therapy offers a promising new avenue for treating CP, generating hope for affected individuals.
Purpose of the Study:
- To systematically review and meta-analyze the efficacy and safety of stem cell treatment in children diagnosed with cerebral palsy.
- Evaluate the impact of stem cell therapy on gross motor function in pediatric CP patients.
Main Methods:
- Conducted a systematic literature search of randomized controlled trials (RCTs) on PubMed and EMBASE.
- Performed a random-effects meta-analysis on 5 RCTs involving 282 patients (0.5-35 years) to assess changes in gross motor function.
- Quantified outcomes using the Gross Motor Function Measure and heterogeneity via the I-squared measure.
Main Results:
- A significant improvement in gross motor function was observed in the stem cell group (SMD 0.95, CI: 0.13-1.76).
- High heterogeneity (I² = 90.1%) was noted across the included studies.
- Serious adverse events were infrequent and similarly distributed between stem cell and control groups.
Conclusions:
- Stem cell therapy demonstrates a statistically significant positive effect on gross motor function in children with CP compared to standard care.
- The clinical significance of the motor function improvement is currently limited.
- Short-term safety is established, but further high-quality RCTs are required to confirm efficacy and long-term outcomes.
Background:
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.
Aim:
The aim is to evaluate the efficacy and safety of stem cell treatment in children with CP using a systematic review and meta-analysis.
Methods:
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.
Results:
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 (I 2 = 90.1%). Serious adverse events were rare and equally distributed among both intervention and control groups.
Conclusion:
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.
