Back to Search
Start Over
Therapeutic evidence of umbilical cord-derived mesenchymal stem cell transplantation for cerebral palsy: a randomized, controlled trial
- Source :
- Stem Cell Research & Therapy, Stem Cell Research & Therapy, Vol 11, Iss 1, Pp 1-12 (2020)
- Publication Year :
- 2020
- Publisher :
- Springer Science and Business Media LLC, 2020.
-
Abstract
- Background Cerebral palsy (CP) is a syndrome of childhood movement and posture disorders. Clinical evidence is still limited and sometimes inconclusive about the benefits of human umbilical cord mesenchymal stem cells (hUC-MSCs) for CP. We conducted a randomized trial to evaluate the safety and efficacy of hUC-MSC transplantation concomitant with rehabilitation in patients with CP. Methods Eligible patients were allocated into the hUC-MSC group and control group. In addition to rehabilitation, the patients in the hUC-MSC group received four transfusions of hUC-MSCs intravenously, while the control group received a placebo. Adverse events (AEs) were collected for safety evaluation in the 12-month follow-up phase. Primary endpoints were assessed as activities of daily living (ADL), comprehensive function assessment (CFA), and gross motor function measure (GMFM) scales. In addition, cerebral metabolic activity was detected by 18F-FDG-PET/CT to explore the possible mechanism of the therapeutic effects. Primary endpoint data were analyzed by ANOVA using SPSS version 20.0. Results Forty patients were enrolled, and 1 patient withdrew informed consent. Therefore, 39 patients received treatments and completed the scheduled assessments. No significant difference was shown between the 2 groups in AE incidence. Additionally, significant improvements in ADL, CFA, and GMFM were observed in the hUC-MSC group compared with the control group. In addition, the standard uptake value of 18F-FDG was markedly increased in 3 out of 5 patients from the hUC-MSC group at 12 months after transplantation. Conclusions Our clinical data showed that hUC-MSC transplantation was safe and effective at improving the gross motor and comprehensive function of children with CP when combined with rehabilitation. Recovery of cerebral metabolic activity might play an essential role in the improvements in brain function in patients with CP. The therapeutic window, transfusion route, and dosage in our study were considerable for reference in clinical application. Trial registration Chictr.org.cn, ChiCTR1800016554. Registered 08 June 2018—retrospectively registered. The public title was “Randomized trial of umbilical cord-derived mesenchymal stem cells for cerebral palsy.”
- Subjects :
- Male
medicine.medical_specialty
medicine.medical_treatment
Medicine (miscellaneous)
Mesenchymal Stem Cell Transplantation
Placebo
Biochemistry, Genetics and Molecular Biology (miscellaneous)
Cerebral palsy
law.invention
lcsh:Biochemistry
Double-Blind Method
Randomized controlled trial
law
Internal medicine
medicine
Clinical endpoint
Humans
lcsh:QD415-436
Umbilical cord
lcsh:R5-920
Rehabilitation
business.industry
Cerebral Palsy
Research
Therapeutic effect
Activities of daily living
Mesenchymal Stem Cells
Cell Biology
medicine.disease
Clinical trial
Transplantation
Child, Preschool
Molecular Medicine
Female
Cell transplantation
lcsh:Medicine (General)
business
Subjects
Details
- ISSN :
- 17576512
- Volume :
- 11
- Database :
- OpenAIRE
- Journal :
- Stem Cell Research & Therapy
- Accession number :
- edsair.doi.dedup.....38aad384ec90a08c2eb90c8e034dd320