Authors: Nguyễn Thanh Liêm (1), Nguyễn Anh Tuấn (1), Vũ Duy Chinh (2), Ngô Văn Đoan (3), Bùi Việt Anh (1)
Vinmec Stem Cell and Gene Technology Research Institute, Hanoi, Vietnam
Department of Rehabilitation, Vinmec International General Hospital, Hanoi, Vietnam
Department of Radiology, Vinmec International General Hospital, Hanoi, Vietnam
Corresponding Author: Nguyễn Thanh Liêm
Journal: BMC Pediatrics 2017; 17:104
Published on: 2017-04-12
Link: https://bmcpediatr.biomedcentral.com/track/pdf/10.1186/s12887-017-0859-z
Background: Stem cell therapy has emerged as a promising approach to improve motor function in patients with cerebral palsy. The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow mononuclear cell transplantation for patients with cerebral palsy related to cerebral hypoxia.
Methods: An open-label, non-controlled clinical trial was conducted at Vinmec International General Hospital. The therapy consisted of two stem cell infusions, one at baseline and another three months later. Disease progression was monitored at three and six months after the first stem cell transplantation, using the Gross Motor Function Measure (GMFM) and the Modified Ashworth Scale to assess muscle tone.
Results: No serious complications were reported during the study. After the stem cell transplantation, 12 patients experienced fever but did not have infections, and 9 patients experienced vomiting, which was easily managed with medication. The gross motor function of the patients significantly improved at 3 and 6 months after transplantion compared to baseline. Both the total score and the scores in each domain of the GMFM-88 and the percentage of GMFM-66 after transplantation increased significantly (p < 0.001). Muscle spasticity also decreased significantly after stem cell transplantation (p < 0.001). This therapy has therapeutic results regardless of gender, age, and GMFCS level (p > 0.05).
Conclusion: Autologous bone marrow mononuclear cell transplantation is a safe and effective therapy for patients with cerebral palsy.
The clinical trial was registered at ClinicalTrials.gov, registration number: NCT02569775 on October 15, 2015.
