The safety and efficacy of umbilical cord blood mononuclear cells in individuals with spastic cerebral palsy: a randomized double-blind sham-controlled clinical trial
Morteza Zarrabi, Masood Ghahvechi Akbari, Man Amanat, Anahita Majmaa, Ali Reza Moaiedi, Hadi Montazerlotfelahi, Masoumeh Nouri, Amir Ali Hamidieh, Reza Shervin Badv, Hossein Karimi, Ali Rabbani, Ali Mohebbi, Shahram Rahimi‐Dehgolan, Rosa Rahimi, Ensieh Dehghan, Massoud Vosough, Saeed Abroun, Farhad Mahvelati Shamsabadi, Ali Reza Tavasoli, Houman Alizadeh, Neda Pak, Gholam Reza Zamani, Mahmoud Mohammadi, Mohsen Javadzadeh, Mohammad Ghofrani, Seyed Hossein Hassanpour, Morteza Heidari, Mohammad Mehdi Taghdiri, Mohamad Javad Mohseni, Zahra Noparast, Safdar Masoomi, Mehrdad Goudarzi, Masood Mohamadpour, Razieh Shodjaee, Solaleh Samimi, Monireh Mohammad, Mona Gholami, Nahid Vafaei, Leyli Koochakzadeh, Amir Valizadeh, Reza Azizi Malamiri, Mahmoud Reza Ashrafi
Abstract
INTRODUCTION: The current multi-center, randomized, double-blind study was conducted among children with cerebral palsy (CP) to assess the safety and efficacy of umbilical cord blood mononuclear cell (UCB-MNC). We performed the diffusion tensor imaging to assess the changes in the white matter structure. METHODS: /kg) UCB-MNCs were administered via intrathecal route in experimental group. The changes in gross motor function measure (GMFM)-66 from baseline to one year after treatment were the primary endpoints. The mean changes in modified Ashworth scale (MAS), pediatric evaluation of disability inventory (PEDI), and CP quality of life (CP-QoL) were also evaluated and compared between groups. The mean changes in fractional anisotropy (FA) and mean diffusivity (MD) of corticospinal tract (CST) and posterior thalamic radiation (PTR) were the secondary endpoints. Adverse events were safety endpoint. RESULTS: There were 72 included individuals (36 cases in each group). The mean GMFM-66 scores increased in experimental group; compared to baseline (+ 9.62; 95%CI: 6.75, 12.49) and control arm (β: 7.10; 95%CI: 2.08, 12.76; Cohen's d: 0.62) and mean MAS reduced in individuals treated with UCB-MNCs compared to the baseline (-0.87; 95%CI: -1.2, -0.54) and control group (β: -0.58; 95%CI: -1.18, -0.11; Cohen's d: 0.36). The mean PEDI scores and mean CP-QoL scores in two domains were higher in the experimental group compared to the control. The imaging data indicated that mean FA increased and MD decreased in participants of UCB-MNC group indicating improvements in white matter structure. Lower back pain, headaches, and irritability were the most common adverse events within 24 h of treatment that were related to lumbar puncture. No side effects were observed during follow-up. CONCLUSIONS: This trial showed that intrathecal injection of UCB-MNCs were safe and effective in children with CP. TRIAL REGISTRATION: The study was registered with ClinicalTrials.gov ( NCT03795974 ).