Cord Blood Treatment for Children With Cerebral Palsy: Individual Participant Data Meta-Analysis.

Abstract

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.