학술논문
Clinical and imaging outcomes after intrathecal injection of umbilical cord tissue mesenchymal stem cells in cerebral palsy: a randomized double-blind sham-controlled clinical trial.
Document Type
Article
Author
Amanat, Man; Majmaa, Anahita; Zarrabi, Morteza; Nouri, Masoumeh; Akbari, Masood Ghahvechi; Moaiedi, Ali Reza; Ghaemi, Omid; Zamani, Fatemeh; Najafi, Sharif; Badv, Reza Shervin; Vosough, Massoud; Hamidieh, Amir Ali; Salehi, Mona; Montazerlotfelahi, Hadi; Tavasoli, Ali Reza; Heidari, Morteza; Mohebi, Hossein; Fatemi, Ali; Garakani, Amir; Ashrafi, Mahmoud Reza
Source
Subject
*INTRATHECAL injections
*MESENCHYMAL stem cells
*TREATMENT effectiveness
*CEREBRAL palsy
*UMBILICAL cord
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Language
ISSN
1757-6512
Abstract
Background: This study assessed the safety and efficacy of intrathecal injection of umbilical cord tissue mesenchymal stem cells (UCT-MSC) in individuals with cerebral palsy (CP). The diffusion tensor imaging (DTI) was performed to evaluate the alterations in white-matter integrity. Methods: Participants (4–14 years old) with spastic CP were assigned in 1:1 ratio to receive either UCT-MSC or sham procedure. Single-dose (2 × 107) cells were administered in the experimental group. Small needle pricks to the lower back were performed in the sham-control arm. All individuals were sedated to prevent awareness. The primary endpoints were the mean changes in gross motor function measure (GMFM)-66 from baseline to 12 months after procedures. The mean changes in the modified Ashworth scale (MAS), pediatric evaluation of disability inventory (PEDI), and CP quality of life (CP-QoL) were also assessed. Secondary endpoints were the mean changes in fractional anisotropy (FA) and mean diffusivity (MD) of corticospinal tract (CST) and posterior thalamic radiation (PTR). Results: There were 36 participants in each group. The mean GMFM-66 scores after 12 months of intervention were significantly higher in the UCT-MSC group compared to baseline (10.65; 95%CI 5.39, 15.91) and control (β 8.07; 95%CI 1.62, 14.52; Cohen's d 0.92). The increase was also seen in total PEDI scores (vs baseline 8.53; 95%CI 4.98, 12.08; vs control: β 6.87; 95%CI 1.52, 12.21; Cohen's d 0.70). The mean change in MAS scores after 12 months of cell injection reduced compared to baseline (−1.0; 95%CI −1.31, −0.69) and control (β −0.72; 95%CI −1.18, −0.26; Cohen's d 0.76). Regarding CP-QoL, mean changes in domains including friends and family, participation in activities, and communication were higher than the control group with a large effect size. The DTI analysis in the experimental group showed that mean FA increased (CST 0.032; 95%CI 0.02, 0.03. PTR 0.024; 95%CI 0.020, 0.028) and MD decreased (CST −0.035 × 10-3; 95%CI −0.04 × 10-3, −0.02 × 10-3. PTR −0.045 × 10-3; 95%CI −0.05 × 10-3, −0.03 × 10-3); compared to baseline. The mean changes were significantly higher than the control group. Conclusions: The UCT-MSC transplantation was safe and may improve the clinical and imaging outcomes. Trial registration: The study was registered with ClinicalTrials.gov (NCT03795974). [ABSTRACT FROM AUTHOR]