Abstract

BackgroundQuality of life (QOL) is an important factor in evaluating the effectiveness of treatment in children with cerebral palsy (CP). The aim of this study was to evaluate the effects of autologous bone marrow mononuclear cells (BM MNCs) on the QOL of children with CP.MethodsFrom December 2015 to December 2016, 30 children with CP aged from 2 to 15 years received two intrathecal infusions of BM MNCs, one at baseline and the other 3 months later, at Vinmec International Hospital. The motor function and muscle tone of the patients were evaluated using the Gross Motor Function Measure (GMFM)-88 and Modified Ashworth Score, respectively. Their QOL was assessed at baseline and 6 months after the first BM MNC transplant using the Vietnamese version of the Cerebral Palsy Quality of Life Questionnaire for children (CP QOL-Child)–the parental proxy report, which comprises seven domains. Nineteen mothers (mean age: 32.9±4.9 years) and 11 fathers (mean age: 36.1±6.8 years) were invited to complete the CP QOL-Child assessment before and after the transplantations, Paired t-tests and multivariate regression analyses were used to evaluate the changes in QOL and GMFM scores and to identify the key factors correlated with the QOL score.ResultsSignificant changes were observed in the children’s gross motor function and muscle spasticity, as evidenced by the GMFM-88 total score, scores for each of its domains, the GMFM-66 percentile and the muscle tone (P < 0.001). Six months after the transplantations, the QOL scores of children with CP were markedly increased (P < 0.001) for all the domains, except for the domain of access to services. In the multivariate regression analysis, significant associations were found between higher age of children and higher QOL except for feeling about functioning and pain and impact of disability domains. Gross Motor Function Classification System (GMFCS) level was negatively correlated with the score of pain and impact of disability domain, while the GMFM-88 scores were positively correlated with the QOL in terms of feelings about functioning and family health domain (P < 0.05).ConclusionThe QOL of the children with CP was noticeably improved 6 months after BM MNC transplantation and was accompanied by improvements in gross motor function and muscle tone.Trial registrationClinicalTrials.gov Identifier: NCT02574923. Registered on October 14, 2015.

Highlights

  • Quality of life (QOL) is an important factor in evaluating the effectiveness of treatment in children with cerebral palsy (CP)

  • The aim of this study was to evaluate the effects of autologous BM Bone marrow mononuclear cells (MNCs) transplantation on QOL and motor function in children with CP

  • The QOL of children with CP was assessed at baseline and 6 months after the first bone marrow mononuclear cells (BM MNCs) transplant using the CP QOL-Child

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Summary

Introduction

Quality of life (QOL) is an important factor in evaluating the effectiveness of treatment in children with cerebral palsy (CP). Cerebral palsy (CP) is a group of permanent movement and posture development disorders [1] and one of the most common causes of motor disability in children [2,3,4,5] with an estimated overall prevalence of 2.11 per 1000 live births [6]. Stem cell transplantation has consistently been shown to improve motor function and decrease muscle spasticity in children with CP [14, 17,18,19] The transplantation of autologous bone marrow mononuclear cells (BM MNCs) improved the development of children with CP [15]. The evaluation of QOL has been used as an approach to achieve insight into a child’s life, to examine aspects of life that are supportive or detrimental and to provide information on the development of interventions [21]

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