脐血来源干细胞治疗早产儿疾病临床研究进展与挑战

doi:10.12372/jcp.2023.23e0634

Abstract

Abstract:

Despite great progress in the treatment of neonatal diseases, complications of preterm infants such as bronchopulmonary dysplasia, perinatal brain injury and necrotizing enterocolitis are still leading causes of neonatal mortality and long term morbidity. Exploring more effective therapies for the prevention or treatment for the complications of preterm infants are the concerns in neonatology. Stem cell therapy especially umbilical cord blood transplantation, have been explored as a new therapeutic option in premature infant diseases, with very promising preclinical and clinical results over the past 20 years. This article outlined the clinical progress, prospects and challenges of stem cells derived from umbilical cord blood in preterm diseases.

Key words: cell therapy, umbilical cord blood-derived stem cell, preterm infant, clinical research

LI Fang, WANG Li. Progress and challenges in clinical research of umbilical cord blood transplantation for the treatment of premature infant diseases[J].Journal of Clinical Pediatrics, 2023, 41(10): 646-653.

Figures/Tables 1

疾病	研究阶段	移植途径及时机	干细胞类型/来源	结论	参考文献
BPD	I期	气管内、生后7~14 d	同种异体hUCB-MSCs、单次、低剂量（1×10⁷/kg）或高剂量（2×10⁷/kg）	近期（移植后84 d）、远期（校正GA 2岁）安全	Chang等^[29]
	I期	气管内、生后5~14 d	同种异体hUCB-MSCs、单次、低剂量（1×10⁷/kg）或高剂量（2×10⁷/kg）	移植后84 d内无移植相关不良事件	Powell等^[30]
	I期	静脉、诊断BPD后	同种异体hUCB-MSCs、单次、6例低剂量组（1×10⁶/kg）、7例高剂量组（5×10⁶/kg）	移植后28 d内无移植相关不良事件	Xia等^[31]
	I期	静脉、诊断BPD后	同种异体hUCB-MSCs、2剂（每剂1×10⁶/kg）	移植后1年无移植相关不良事件	Nguyen等^[32]
	II期	气管内、生后（11.8± 2.0）d	同种异体hUCB-MSCs、单次1×10⁷/kg	干细胞组炎症因子水平较低、死亡/中重度BPD无显著差异；亚组分析23~24周组重度BPD较低	Ahn等^[33]
	II期	静脉、生后8 h内	自体脐带血单个核细胞、单次5×10⁷ /kg	干细胞组机械通气和需氧治疗时间显著减少	Ren等^[34]
	II期	静脉、生后24 h内	自体脐带血单个核细胞、单次5×10⁷ /kg	干细胞组存活者的中重度BPD发病率显著降低、呼吸机拔管率更高、校正18~24月龄发育迟缓更低	Zhu xiao等^[35]
IVH	I期	脑室内、诊断重度IVH后7 d内	同种异体hUCB-MSCs、单次、低剂量（5×10⁶/kg）或高剂量（2×10⁷/kg）	近期结果：安全可行；计划随访至2岁	Ahn等^[38]
HIE	I期	静脉、生后最初72（48）h内最多输注4次	（1~5）×10⁷ /kg自体脐带血	住院结局相似、干细胞移植组1岁Bayley III评分更好	Cotton等^[39]
HIE	I期	静脉、生后12~72 h内共接受3次	自体脐带血	随访至18月龄安全可行	Tsuji等^[40]
NEC	个案报道	静脉、NEC术后	同种异体hUCB-MSCs 1×10⁷/kg	1岁的体格和神经发育和同龄儿童相当	Akduman等^[42]
早产儿贫血	I期	静脉	5例、自体脐带血移植	安全可行	Rudnicki等^[28]
早产儿贫血	I期	静脉	5例、自体脐带血移植	安全可行	Kotowski等^[43]
左心发育不良综合征	I期	心肌内注射、接受II期手术时	自体脐带血单个核细胞（1~3）×10⁶	随访6个月：安全可行	Burkhar等^[44]

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Progress and challenges in clinical research of umbilical cord blood transplantation for the treatment of premature infant diseases

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