Preclinical and early phase clinical studies suggest that an appropriately dosed umbilical cord blood (CB) infusion has the potential to help improve motor function in young children with cerebral palsy (CP). As many children with CP do not have their own CB available, use of allogeneic cells would extend access to this potentially beneficial therapy to more children. In this phase I, open-label study, 15 children, aged 1 to 6 years, with moderate to severe spastic CP were treated with a single intravenous infusion of allogeneic human leukocyte antigen (HLA) matched or partially matched sibling CB with a cell dose of ≥2.5 × 10⁷ cells/kg based on the pre-cryopreservation count (median infused cell dose, 3.3 × 10⁷; range, 1.8-5.2 × 10⁷). There were a total of 49 adverse events (AEs) over a 2-year time period, but there were no AEs related to the CB infusions. Specifically, there were no acute infusion reactions and no antibody formation against platelets, red blood cells, or donor-specific HLA antigens. Donor cells were not detected in peripheral blood 6 months later. Six months after infusion, participants were assessed for response and experienced a mean ± SD increase of 4.7 ± 2.5 points on the Gross Motor Function Measure-66 and 1 ± 2.9 points on the Peabody Gross Motor Quotient. Appropriately dosed, allogeneic partially or fully HLA-matched sibling CB infusion is well tolerated and potentially beneficial in young children with CP.

中文翻译：

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脑瘫幼儿的兄弟姐妹脐带血输注是安全的

临床前和早期临床研究表明，适当剂量的脐带血 (CB) 输注有可能帮助改善脑瘫 (CP) 幼儿的运动功能。由于许多患有 CP 的儿童没有自己的 CB，因此使用同种异体细胞将使更多儿童获得这种潜在有益的治疗。在这项 I 期开放标签研究中，15 名 1 至 6 岁的中度至重度痉挛性 CP 患儿接受了单次静脉输注同种异体人类白细胞抗原 (HLA) 匹配或部分匹配的同胞 CB 与细胞剂量的单次静脉输注≥2.5 × 10 ^{7 个}细胞/kg，基于预冻存计数（中位输注细胞剂量，3.3 × 10 ⁷；范围，1.8-5.2 × 10 ⁷）。在 2 年的时间段内共有 49 起不良事件 (AE)，但没有与 CB 输注相关的 AE。具体来说，没有急性输注反应，也没有针对血小板、红细胞或供体特异性 HLA 抗原的抗体形成。6 个月后在外周血中未检测到供体细胞。输注 6 个月后，对参与者的反应进行了评估，并在粗大运动功能测量 66 上的平均 ± SD 增加了 4.7 ± 2.5 分，在皮博迪粗大运动商数上增加了 1 ± 2.9 分。适当剂量的同种异体部分或完全 HLA 匹配的同胞 CB 输注对患有 CP 的幼儿具有良好的耐受性和潜在的益处。