间充质基质细胞(MSCs)是人体所有器官中罕见的前体细胞。MSC在体外和体内具有深远的抗炎作用并降低同种异体反应性。在小儿异基因造血细胞移植(HCT)中,MSCs主要用于治疗急性移植物抗宿主病(GVHD)。在加拿大,MSCs可用于该适应症,Japan,和新西兰。儿科患者中MSCs的更罕见的适应症包括移植失败和慢性GVHD。骨髓间充质干细胞,脂肪组织,脐带,沃顿的果冻,胎盘组织,decidua已经被使用了,但最佳临床基质细胞来源尚未在临床试验中进行比较。使用MSCs的更多实验临床适应症,比如败血症,急性呼吸窘迫综合征,出血,肺纵隔,和神经炎症主要在动物模型或成人HCT患者中进行了研究。MSC几乎没有任何副作用。在这项初步研究中,我们报告了六个接受蜕膜基质细胞(DSC)治疗类固醇难治性急性GVHD的儿童的结果。6个月时,4例患者出现完全缓解,2例患者出现部分缓解.一名高风险ALL儿童死于复发,一名镰状细胞病男孩死于脑出血。5年生存率为67%,所有幸存者的Lansky评分为100%。最后,来自各种器官的MSC具有良好的耐受性,并且在儿科患者中对急性GVHD表现出令人鼓舞的结果。
Mesenchymal stromal cells (MSCs) are rare precursors in all organs of the body. MSCs have profound anti-inflammatory effects and reduce alloreactivity in vitro and in vivo. In pediatric allogeneic hematopoietic cell transplantation (HCT), MSCs have mainly been used to treat acute graft-versus-host disease (GVHD). MSCs are commercially available for this indication in Canada, Japan, and New Zeeland. More rare indications for MSCs in pediatric patients include graft failure and chronic GVHD. MSCs from bone marrow, adipose tissue, umbilical cord, Wharton\'s jelly, placenta tissue, and decidua have been used, but the optimal clinical stromal cell source has not been compared in clinical trials. More experimental clinical indications using MSCs, such as sepsis, acute respiratory distress syndrome, hemorrhages, pneumo-mediastinum, and neuroinflammation have primarily been explored in animal models or adult HCT patients. MSCs have almost no if any side-effects. In this pilot study we report the outcome of six children treated with decidua stromal cells (DSCs) for steroid refractory acute GVHD. At 6 months, complete response was seen in four patients and partial response in two patients. One child with high-risk ALL died from relapse and a boy with sickle cell disease died from a cerebral hemorrhage. Five-year survival was 67% and all survivors showed a Lansky score of 100%. To conclude, MSCs from various organs are well-tolerated and have shown an encouraging outcome for acute GVHD in pediatric patients.