Brain & development · 2025
Safety and feasibility of allogeneic sibling cord blood infusion in Japanese children with cerebral palsy: A single-center pilot study.
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Saitoh Shiho, Kikuchi Hiroaki, Marukane Takuzo, Tsuno Terumasa, Hosoda Rina, Baba Nobuyasu, Wang Feifei, Kuroiwa Yumi, Nagai Ryuhei, Tsuda Masayuki, Maeda Nagamasa, Sagara Yusuke, Fujieda Mikiya
Аннотация
INTRODUCTION: Cerebral palsy (CP) is the most common motor disorder in childhood that causes lifelong disabilities. Studies suggest that administration of autologous cord blood (CB) or sibling cord blood (SCB) may improve gross motor function and brain connectivity in CP. In this pilot study, we evaluated the safety and efficacy of allogeneic SCB in Japanese children with CP.
METHODS: A single-arm pilot study of a single intravenous dose of HLA-matched or partially matched (at least 4/6 HLA molecular matches) SCB was conducted in five Japanese patients with CP (5.0-6.3 years old). The primary endpoint was to measure the safety profiles and assessment of motor function and neurodevelopmental behaviors as the secondary endpoint.
RESULTS: No serious side effects or concerning infusion reactions were observed. All patients showed a mean improvement of 4.89 ± 4.01 points in Gross Motor Function Measure-66 (GMFM-66) score at 6 months, which was better than predicted by age and severity of disease. In addition, GMFM-66 scores at 1 and 2 years was further improved by a mean of 6.49 ± 3.58 and 7.93 ± 4.26 points, respectively. One case showed improvement in the Developmental Quotient (DQ) overall and language/social scales and another case showed improvement in the Wechsler Intelligence Scale for Children, Fourth Edition (WISC-IV) overall, language. No significant correlations were found between GMFM-66 scores, DQ scales, WISC-IV scores and HLA concordance or number of cells administered.
CONCLUSION: Allogeneic SCB administration is safe and feasible in Japanese CP patients and may offer therapeutic potential in clinical practice. Future study is needed to clarify several unclear issues.
METHODS: A single-arm pilot study of a single intravenous dose of HLA-matched or partially matched (at least 4/6 HLA molecular matches) SCB was conducted in five Japanese patients with CP (5.0-6.3 years old). The primary endpoint was to measure the safety profiles and assessment of motor function and neurodevelopmental behaviors as the secondary endpoint.
RESULTS: No serious side effects or concerning infusion reactions were observed. All patients showed a mean improvement of 4.89 ± 4.01 points in Gross Motor Function Measure-66 (GMFM-66) score at 6 months, which was better than predicted by age and severity of disease. In addition, GMFM-66 scores at 1 and 2 years was further improved by a mean of 6.49 ± 3.58 and 7.93 ± 4.26 points, respectively. One case showed improvement in the Developmental Quotient (DQ) overall and language/social scales and another case showed improvement in the Wechsler Intelligence Scale for Children, Fourth Edition (WISC-IV) overall, language. No significant correlations were found between GMFM-66 scores, DQ scales, WISC-IV scores and HLA concordance or number of cells administered.
CONCLUSION: Allogeneic SCB administration is safe and feasible in Japanese CP patients and may offer therapeutic potential in clinical practice. Future study is needed to clarify several unclear issues.