Department of Pediatrics, New York Medical College, Valhalla, NY 10595, USA.
Bone Marrow Transplant. 2013 Jul;48(7):890-900. doi: 10.1038/bmt.2012.169. Epub 2012 Sep 10.
Brain injury resulting from perinatal hypoxic-ischemic encephalopathy (HIE) is a major cause of acute mortality in infants and chronic neurologic disability in surviving children. Recent multicenter clinical trials demonstrated the effectiveness of hypothermia initiated within the first 6 postnatal hours to reduce the risk of death or major neurological disabilities among neonates with HIE. However, in these trials, approximately 40% of cooled infants died or survived with significant impairments. Therefore, adjunct therapies are required to improve the outcome in neonates with HIE. Cord blood (CB) is a rich source of stem cells. Administration of human CB cells in animal models of HIE has generally resulted in improved outcomes and multiple mechanisms have been suggested including anti-inflammation, release of neurotrophic factors and stimulation of endogenous neurogenesis. Investigators at Duke are conducting studies of autologous CB infusion in neonates with HIE and in children with cerebral palsy. These pilot studies indicate no added risk from the regimens used, but results of ongoing placebo-controlled trials are needed to assess efficacy. Meanwhile, further investigations are warranted to determine the best strategies, that is, timing, dosing, route of delivery, choice of stem cells and ex vivo modulations, to attain long-term benefits of CB stem cell therapy.
围产期缺氧缺血性脑病（HIE）导致的脑损伤是导致婴儿急性死亡和存活儿童慢性神经功能障碍的主要原因。最近的多中心临床试验表明，在出生后 6 小时内开始降温可降低 HIE 新生儿死亡或发生重大神经功能障碍的风险。然而，在这些试验中，约 40%的接受降温治疗的婴儿死亡或存活，但存在严重的损害。因此，需要辅助治疗来改善 HIE 新生儿的预后。脐带血（CB）是干细胞的丰富来源。在 HIE 的动物模型中给予人 CB 细胞通常会改善结局，并且已经提出了多种机制，包括抗炎、释放神经营养因子和刺激内源性神经发生。杜克大学的研究人员正在对 HIE 新生儿和脑瘫儿童进行自体 CB 输注的研究。这些初步研究表明，所用方案没有增加风险，但需要进行正在进行的安慰剂对照试验来评估疗效。同时，需要进一步的研究来确定最佳策略，即时间、剂量、给药途径、干细胞选择和体外调节，以实现 CB 干细胞治疗的长期获益。
