The effects of bone marrow mononuclear cell transplantation on the quality of life of children with cerebral palsy

Thanh Liem Nguyen, Hoang Phuong Nguyen, Trung Kien Nguyen, Thanh Liem Nguyen, Hoang Phuong Nguyen, Trung Kien Nguyen

Abstract

Background: 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 bone marrow mononuclear cells (BM MNCs) on the QOL of children with CP.

Methods: From 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.

Results: Significant 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).

Conclusion: The 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 registration: ClinicalTrials.gov Identifier: NCT02574923 . Registered on October 14, 2015.

Keywords: Cerebral palsy; Quality of life; Stem cell transplantation.

Conflict of interest statement

Ethics approval and consent to participate

The study was approved by the Vinmec International Hospital Ethics Committee under reference number 382/2015/QD-VINMEC. The committee evaluated the ethical aspects of the study in accordance with the World Medical Association Declaration of Helsinki. All caregivers received a written consent form, a cover letter and a clear explanation of the safety issues, potential risks and benefits, and the procedure involved.

Consent for publication

Parental written informed consent was obtained well before patient enrollment in every case. This consent included their agreement about publishing the indirect identifiers of the patients such as age and gender.

Competing interests

The authors declare that they have no competing interests.

Publisher’s Note

Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

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