Panda Prateek Kumar, Kasinathan Ananthanarayanan, Panda Pragnya, Dawman Lesa, Gupta Aditi, Malik Vivek Singh, Sharawat Indar Kumar, Singh Meenu
Pediatric Neurology Division, Department of Pediatrics, All India Institute of Medical Sciences, Rishikesh, Uttarakhand, India, 249203.
Department of Pediatric Neurology, Rainbow Children's Hospital, Chennai, Tamilnadu, India.
Childs Nerv Syst. 2025 Aug 22;41(1):265. doi: 10.1007/s00381-025-06931-0.
Apart from therapeutic hypothermia, no treatment is approved for neonates with hypoxic-ischemic encephalopathy (HIE). Stem cell therapy is a promising option, with a few studies conducted in recent years.
This systematic review assessed pooled 12-month survival and neurodevelopmental outcomes in neonates with HIE receiving stem cell therapy. Secondary outcomes included survival with favorable neurodevelopment, seizures during hospitalization, discharge on antiseizure medication (ASM), and 100% oral feeding. Both controlled and uncontrolled trials were included.
Four studies, including one quadruple-blinded randomized-controlled trial (RCT), met the inclusion criteria, comprising 153 neonates, with 52 in the stem cell therapy group and 101 in the standard care group. Among those receiving stem cell therapy, 46 neonates received umbilical cord blood-derived stem cells, while 6 received human cord tissue mesenchymal stem cells. The pooled 12-month survival rate in the stem cell group was 92% (95% CI: 82%-98%, I = 0%), and survival with Bayley scores of 85 or more in all three domains was 76% (95% CI: 62%-87%, I = 0%). Survival with favorable neurodevelopmental outcomes was significantly higher in the stem cell therapy group (RR = 1.89, 95% CI: 1.30-2.74, I = 0%, p = 0.0008). However, overall survival at 12 months (RR = 1.09, 95% CI: 0.94-1.26, p = 0.25), seizures during hospitalization (RR = 0.76, 95% CI: 0.41-1.41, p = 0.38), discharge on ASM (RR = 0.83, 95% CI: 0.35-1.97, p = 0.67), and adverse events were comparable between groups.
Stem cell therapy appears safe and may improve neurodevelopmental outcomes in neonates with HIE. However, larger, more robust RCTs are needed before universal recommendations can be made.
除治疗性低温外，目前尚无获批用于治疗新生儿缺氧缺血性脑病（HIE）的疗法。干细胞疗法是一种有前景的选择，近年来已有一些相关研究。
本系统评价评估了接受干细胞治疗的HIE新生儿的12个月合并生存率和神经发育结局。次要结局包括神经发育良好的生存情况、住院期间的癫痫发作、出院时使用抗癫痫药物（ASM）以及完全经口喂养。纳入了对照试验和非对照试验。
四项研究符合纳入标准，其中包括一项四盲随机对照试验（RCT），共153例新生儿，干细胞治疗组52例，标准治疗组101例。在接受干细胞治疗的患儿中，46例接受了脐带血来源的干细胞，6例接受了人脐带组织间充质干细胞。干细胞组的12个月合并生存率为92%（95%CI：82%-98%，I² = 0%），贝利量表所有三个领域得分均达到85分及以上的生存率为76%（95%CI：62%-87%，I² = 0%）。干细胞治疗组神经发育结局良好的生存率显著更高（RR = 1.89，95%CI：1.30-2.74，I² = 0%，p = 0.0008）。然而，两组在12个月时的总体生存率（RR = 1.09，95%CI：0.94-1.26，p = 0.25）、住院期间的癫痫发作（RR = 0.76，95%CI：0.41-1.41，p = 0.38）、出院时使用ASM（RR = 0.83，95%CI：0.35-1.97，p = 0.67）以及不良事件方面相当。
干细胞疗法似乎是安全的，可能改善HIE新生儿的神经发育结局。然而，在做出普遍推荐之前，需要开展更大规模、更有力的随机对照试验。
