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Cord Blood Treatment for Children With Cerebral Palsy: Individual Participant Data Meta-Analysis
Megan Finch-Edmondson1,2, Madison C B Paton1,2, Annabel Webb1,2
1Cerebral Palsy Alliance Research Institute, Specialty of Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.
Insights
Umbilical cord blood (UCB) therapy shows promise for improving gross motor function in children with cerebral palsy (CP). Higher UCB doses and younger children with milder CP experienced greater benefits, with UCB treatment being safe.
Area of Science:
- Regenerative Medicine
- Neuroscience
- Pediatric Neurology
Background:
- Umbilical cord blood (UCB) is an emerging therapy for cerebral palsy (CP).
- Previous trials suggest UCB can enhance gross motor function in children with CP.
- Heterogeneity in study designs has previously limited definitive interpretation of UCB's efficacy.
Purpose of the Study:
- To evaluate the safety and effectiveness of UCB for improving gross motor function in children diagnosed with CP.
- To investigate the relationship between UCB cell dose and treatment outcomes.
- To identify subgroups of children with CP who may respond best to UCB therapy.
Main Methods:
- A systematic search identified relevant published reports and registered trials.
- Individual participant data from 11 studies were pooled for a 1-stage meta-analysis.
- Linear mixed models were used to analyze the effect of UCB on Gross Motor Function Measure (GMFM) scores, including cell dose and responder subgroup analyses.
Main Results:
- Data from 170 UCB-treated participants and 171 controls revealed a significant improvement in mean GMFM-66 scores at 6 and 12 months post-treatment (P=.005 and P=.012, respectively).
- A dose-dependent increase in GMFM-66 effect size was observed at 3 and 12 months (P<.001 and P=.047, respectively).
- CP severity and younger age (<5 years) were associated with greater GMFM-66 benefits. Serious adverse event rates were comparable between UCB and control groups.
Conclusions:
- UCB therapy is safe and demonstrates efficacy in improving gross motor function for select children with CP.
- Higher UCB doses correlate with enhanced therapeutic effects.
- Future clinical trials can leverage these findings for precision-based designs, targeting younger children with milder CP for optimal outcomes.
Context:
Umbilical cord blood (UCB) is a novel treatment for cerebral palsy (CP), with trials indicating UCB can improve gross motor function. However, heterogeneity has limited the ability to interpret findings.
Objective:
Assess the safety and efficacy of UCB for improving gross motor function in children with CP, including exploring cell dose effect and responder subgroups.
Data Sources:
Individual participant data from published reports and registered trials identified via systematic searches.
Study Selection:
Studies administering UCB to individuals with CP collecting Gross Motor Function Measure (GMFM) scores.
Data Extraction:
A 1-stage individual participant data meta-analysis was conducted in R to obtain the pooled effect of UCB and cell dose effect on GMFM using linear mixed models. Responder subgroups were also investigated.
Results:
Four hundred ninety-eight participant data records were obtained from 11 studies. Main analysis of 170 participants treated with UCB and 171 controls demonstrated UCB increased mean GMFM-66 score compared with controls by 1.36 points at 6 months (95% CI, 0.41-2.32; P = .005) and 1.42 at 12 months (95% CI, 0.31-2.52; P = .012). Mean GMFM-66 effect size increased with increasing cell dose at 3 (P < .001) and 12 months (P = .047). CP severity and baseline age were associated with GMFM-66 effect size. The rate of serious adverse events was similar between groups.
Limitations:
Heterogeneity across variables and time points, reducing subanalysis power.
Conclusions:
UCB is safe and provides benefit for improving gross motor function in some children with CP, with higher doses associated with increased effect size. Younger participants (aged approximately <5 years) with milder CP showed increased benefit. Findings will help design future trials with precision.
