PMID- 35351020
OWN - NLM
STAT- MEDLINE
DCOM- 20220331
LR  - 20231103
IS  - 1471-2377 (Electronic)
IS  - 1471-2377 (Linking)
VI  - 22
IP  - 1
DP  - 2022 Mar 29
TI  - The safety and efficacy of umbilical cord blood mononuclear cells in individuals 
      with spastic cerebral palsy: a randomized double-blind sham-controlled clinical 
      trial.
PG  - 123
LID - 10.1186/s12883-022-02636-y [doi]
LID - 123
AB  - 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: Males and females aged 4 to 14 years old with spastic CP were included. 
      Eligible participants were allocated in 4:1 ratio to be in the experimental or 
      control groups; respectively. Individuals who were assigned in UCB-MNC group were 
      tested for human leukocyte antigen (HLA) and fully-matched individuals were 
      treated with UCB-MNCs. A single dose (5 x 10(6) /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 (beta: 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 (beta: -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 ).
CI  - (c) 2022. The Author(s).
FAU - Zarrabi, Morteza
AU  - Zarrabi M
AD  - Department of Regenerative Medicine, Cell Science Research Center, Royan 
      Institute for Stem Cell Biology and Technology, ACECR, Tehran, Iran.
FAU - Akbari, Masood Ghahvechi
AU  - Akbari MG
AD  - Physical Medicine and Rehabilitation Department, Children's Medical Center, 
      Tehran University of Medical Sciences, Tehran, Iran.
FAU - Amanat, Man
AU  - Amanat M
AD  - Division of Neurogenetics and Neuroscience, The Moser Center for 
      Leukodystrophies, Kennedy Krieger Institute, Johns Hopkins University, Baltimore, 
      MD, USA.
FAU - Majmaa, Anahita
AU  - Majmaa A
AD  - Pediatrics Center of Excellence, Pediatric Intensive Unit, Children's Medical 
      Center, Tehran University of Medical Sciences, Tehran, Iran.
FAU - Moaiedi, Ali Reza
AU  - Moaiedi AR
AD  - Department of Pediatric Neurology, Clinical Research Development Center of 
      Children Hospital, Hormozgan University of Medical Sciences, Bandar Abbas, Iran.
FAU - Montazerlotfelahi, Hadi
AU  - Montazerlotfelahi H
AD  - Department of Pediatrics, Faculty of Medicine, Tehran Medical Sciences, Islamic 
      Azad University, Tehran, Iran.
FAU - Nouri, Masoumeh
AU  - Nouri M
AD  - R & D Department, Royan Stem Cell Technology Co, Tehran, Iran.
FAU - Hamidieh, Amir Ali
AU  - Hamidieh AA
AD  - Pediatrics Center of Excellence Pediatric Hematology, Oncology and Stem Cell 
      Transplantation Department, Children's Medical Center, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Badv, Reza Shervin
AU  - Badv RS
AD  - Pediatrics Center of Excellence, Department of Pediatric Neurology, Children's 
      Medical Center, Growth and Development Research Center, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Karimi, Hossein
AU  - Karimi H
AD  - Neurorehabilitation Research Center University of Welfare and Rehabilitation 
      Sciences, Tehran, Iran.
FAU - Rabbani, Ali
AU  - Rabbani A
AD  - Pediatrics Center of Excellence Pediatric Endocrinology Department, Growth and 
      Development Research Center, Children's Medical Center, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Mohebbi, Ali
AU  - Mohebbi A
AD  - Pediatrics Center of Excellence, Growth and Development Research Center, 
      Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran.
FAU - Rahimi-Dehgolan, Shahram
AU  - Rahimi-Dehgolan S
AD  - Physical Medicine and Rehabilitation Department, Tehran University of Medical 
      Sciences, Tehran, Iran.
FAU - Rahimi, Rosa
AU  - Rahimi R
AD  - Physical Medicine and Rehabilitation Department, Khatamolanbia Hospital, Tehran, 
      Iran.
FAU - Dehghan, Ensieh
AU  - Dehghan E
AD  - Transplantation Department, Royan Stem Cell Technology Co, Tehran, Iran.
FAU - Vosough, Massoud
AU  - Vosough M
AD  - Department of Regenerative Medicine, Cell Science Research Center, Royan 
      Institute for Stem Cell Biology and Technology, ACECR, Tehran, Iran.
FAU - Abroun, Saeed
AU  - Abroun S
AD  - Department of Hematology, Faculty of Medical Sciences, Tarbiat Modares 
      University, Tehran, Iran.
FAU - Shamsabadi, Farhad Mahvelati
AU  - Shamsabadi FM
AD  - Department of Pediatric Neurology, Atieh Hospital, Tehran, Iran.
FAU - Tavasoli, Ali Reza
AU  - Tavasoli AR
AD  - Pediatrics Center of Excellence, Department of Pediatric Neurology, Children's 
      Medical Center, Growth and Development Research Center, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Alizadeh, Houman
AU  - Alizadeh H
AD  - Pediatrics Center of Excellence, Department of Radiology, Children's Medical 
      Center, Tehran University of Medical Sciences, Tehran, Iran.
FAU - Pak, Neda
AU  - Pak N
AD  - Pediatrics Center of Excellence, Department of Radiology, Children's Medical 
      Center, Tehran University of Medical Sciences, Tehran, Iran.
FAU - Zamani, Gholam Reza
AU  - Zamani GR
AD  - Pediatrics Center of Excellence, Department of Pediatric Neurology, Children's 
      Medical Center, Growth and Development Research Center, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Mohammadi, Mahmoud
AU  - Mohammadi M
AD  - Pediatrics Center of Excellence, Department of Pediatric Neurology, Children's 
      Medical Center, Growth and Development Research Center, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Javadzadeh, Mohsen
AU  - Javadzadeh M
AD  - Department of Pediatric Neurology, Mofid Children's Hospital, Pediatric Neurology 
      Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Iran.
FAU - Ghofrani, Mohammad
AU  - Ghofrani M
AD  - Department of Pediatric Neurology, Mofid Children's Hospital, Pediatric Neurology 
      Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Iran.
FAU - Hassanpour, Seyed Hossein
AU  - Hassanpour SH
AD  - Department of Pediatric Neurology, Aliasghar Children's Hospital, Iran University 
      of Medical Sciences, Tehran, Iran.
FAU - Heidari, Morteza
AU  - Heidari M
AD  - Pediatrics Center of Excellence, Department of Pediatric Neurology, Children's 
      Medical Center, Growth and Development Research Center, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Taghdiri, Mohammad Mehdi
AU  - Taghdiri MM
AD  - Department of Pediatric Neurology, Mofid Children's Hospital, Pediatric Neurology 
      Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Iran.
FAU - Mohseni, Mohamad Javad
AU  - Mohseni MJ
AD  - Pediatric Urology Research Center, Pediatrics Center of Excellence, Children's 
      Medical Center, Tehran University of Medical Sciences, Tehran, Iran.
FAU - Noparast, Zahra
AU  - Noparast Z
AD  - Department of Pediatric Nephrology, Bahrami Hospital, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Masoomi, Safdar
AU  - Masoomi S
AD  - Department of Epidemiology and Biostatistics, School of Public Health, Tehran 
      University of Medical Sciences, Tehran, Iran.
FAU - Goudarzi, Mehrdad
AU  - Goudarzi M
AD  - Department of Pediatric Anesthesiology, Children's Medical Center, Tehran 
      University of Medical Sciences, Tehran, Iran.
FAU - Mohamadpour, Masood
AU  - Mohamadpour M
AD  - Pediatrics Center of Excellence, Pediatric Intensive Unit, Children's Medical 
      Center, Tehran University of Medical Sciences, Tehran, Iran.
FAU - Shodjaee, Razieh
AU  - Shodjaee R
AD  - Department of Regenerative Medicine, Cell Science Research Center, Royan 
      Institute for Stem Cell Biology and Technology, ACECR, Tehran, Iran.
FAU - Samimi, Solaleh
AU  - Samimi S
AD  - Physical Medicine and Rehabilitation Department, Khatamolanbia Hospital, Tehran, 
      Iran.
FAU - Mohammad, Monireh
AU  - Mohammad M
AD  - Process Department, Royan Stem Cell Technology Co, Tehran, Iran.
FAU - Gholami, Mona
AU  - Gholami M
AD  - Physical Medicine and Rehabilitation Department, Khatamolanbia Hospital, Tehran, 
      Iran.
FAU - Vafaei, Nahid
AU  - Vafaei N
AD  - Faculty of Medicine, Students' Scientific Research Center, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Koochakzadeh, Leyli
AU  - Koochakzadeh L
AD  - Pediatrics Center of Excellence Pediatric Hematology, Department of Hematology & 
      Oncology, Children's Medical Center, Tehran University of Medical Sciences, 
      Tehran, Iran.
FAU - Valizadeh, Amir
AU  - Valizadeh A
AD  - Faculty of Medicine, Students' Scientific Research Center, Tehran University of 
      Medical Sciences, Tehran, Iran.
FAU - Malamiri, Reza Azizi
AU  - Malamiri RA
AD  - Department of Paediatric Neurology, Golestan Medical, Educational, and Research 
      Center, Ahvaz Jundishapur University of Medical Sciences, Ahvaz, Iran.
FAU - Ashrafi, Mahmoud Reza
AU  - Ashrafi MR
AD  - Pediatrics Center of Excellence, Department of Pediatric Neurology, Children's 
      Medical Center, Growth and Development Research Center, Tehran University of 
      Medical Sciences, Tehran, Iran. ashrafim@tums.ac.ir.
LA  - eng
SI  - ClinicalTrials.gov/NCT03795974
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
DEP - 20220329
PL  - England
TA  - BMC Neurol
JT  - BMC neurology
JID - 100968555
SB  - IM
MH  - Adolescent
MH  - *Cerebral Palsy
MH  - Child
MH  - Child, Preschool
MH  - Diffusion Tensor Imaging/methods
MH  - Double-Blind Method
MH  - Female
MH  - Fetal Blood
MH  - Humans
MH  - Male
MH  - Quality of Life
PMC - PMC8966246
OTO - NOTNLM
OT  - Cerebral palsy
OT  - Children
OT  - Mononuclear cells
OT  - Stem cells
OT  - Umbilical cord
COIS- The authors declare that they have no competing interests.
EDAT- 2022/03/31 06:00
MHDA- 2022/04/01 06:00
PMCR- 2022/03/29
CRDT- 2022/03/30 05:36
PHST- 2021/07/15 00:00 [received]
PHST- 2022/03/15 00:00 [accepted]
PHST- 2022/03/30 05:36 [entrez]
PHST- 2022/03/31 06:00 [pubmed]
PHST- 2022/04/01 06:00 [medline]
PHST- 2022/03/29 00:00 [pmc-release]
AID - 10.1186/s12883-022-02636-y [pii]
AID - 2636 [pii]
AID - 10.1186/s12883-022-02636-y [doi]
PST - epublish
SO  - BMC Neurol. 2022 Mar 29;22(1):123. doi: 10.1186/s12883-022-02636-y.