Abstract

BackgroundStem cell therapy has emerged as a promising method for improving motor function of patients with cerebral palsy. The aim of this study is to assess the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation in patients with cerebral palsy related to oxygen deprivation.MethodsAn open label uncontrolled clinical trial was carried out at Vinmec International Hospital. The intervention consisted of two administrations of stem cells, the first at baseline and the second 3 months later. Improvement was monitored at 3 months and 6 months after the first administration of stem cells, using the Gross Motor Function Measure (GMFM) and Modified Ashworth Score which measures muscle tone.ResultsNo severe complications were recorded during the study. After transplantation, 12 patients encountered fever without infections and 9 patients experienced vomiting which was easily managed with medications. Gross motor function was markedly improved 3 months or 6 months after stem cell transplantation than at baseline. The post-transplantation GMFM-88 total score, each of its domains and the GMFM-66 percentile were all significantly higher (p-value < 0.001). Muscle spasticity also reduced significantly after transplantation (p-value < 0.001). The therapy was equally effective regardless of sex, age and GMFCS level (p-value > 0.05).ConclusionAutologous bone marrow mononuclear cell transplantation appears to be a safe and effective therapy for patients with cerebral palsy.Trial registrationClinicalTrials.gov Identifier: NCT02569775. Retrospectively registered on October 15, 2015.

Highlights

  • Stem cell therapy has emerged as a promising method for improving motor function of patients with cerebral palsy

  • More scientific evidence is needed to provide support for stem cell therapy as a standard of care for cerebral palsy. The aim of this clinical study is to evaluate the safety and effectiveness of autologous bone marrow mononuclear cells (BMMNCs) in the management of cerebral palsy related to oxygen deprivation at Vinmec International Hospital, Hanoi, Vietnam

  • In our study of the 40 patients with cerebral palsy related to oxygen deprivation, transplantation of autologous BMMNCs was apparently safe with no adverse events recorded during the procedure

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Summary

Introduction

Stem cell therapy has emerged as a promising method for improving motor function of patients with cerebral palsy. The aim of this study is to assess the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation in patients with cerebral palsy related to oxygen deprivation. Cerebral palsy is a non-progressive brain disorder affecting movement and posture. Management of cerebral palsy includes physical therapy, neurectomy, botulinum toxin A and medications, these could not cure the disease [2]. The mechanism of action by which stem cells exert their effects in cerebral palsy is still a topic of controversy. Many other studies suggested that it was not the stem cell differentiation that would replace the injured cells but that the stem cells secreted trophic factors and cytokines, which modulate the micro environment, support anti-inflammation, cytoprotection and angiogenesis; generate myelinproducing cells and stimulate endogenous stem cells in the brain [11,12,13,14,15]

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