Steele Ann, Steele Peter
All Children's Hospital, Department of Pathology and Laboratory Medicine, 801 6th Street South, St Petersburg, FL 33701, USA.
Curr Opin Pediatr. 2006 Oct;18(5):518-23. doi: 10.1097/01.mop.0000245352.75830.1d.
Stem cell therapy for treatment of cardiac disease has shown therapeutic potential.
A number of stem and progenitor populations have been identified for potential use in cardiac repair. Each possesses a unique potency that justifies consideration for use. Autologous, unfractionated bone marrow cells or skeletal myoblasts were used in early clinical trails to evaluate reparative effects on recent or record infarcts. In each case, evidence of limited improvement in cardiac function was obtained. Myoblast grafts were unexpectedly correlated with arrhythmias, thereby identifying a safety issue. The small number of patients and the lack of randomized control groups preclude conclusions regarding efficacy. Randomized controlled, intermediate-sized, double-blind clinical trials must be undertaken to this end.
Cellular therapy may be useful in the treatment of cardiac disease in adults. Appropriate adaptations to meet unique requirements for treatment of pediatric cardiovascular disease may be required. Bone marrow and skeletal myoblasts do not promote true tissue regeneration in spite of observed functional improvement. Trials using cells possessing true potential for (trans)differentiation may elucidate the potential and value of this therapy as a reparative modality. Development of optimal strategies for targeted delivery consistent with pathobiology is of exception clinical relevance.
干细胞疗法在治疗心脏病方面已显示出治疗潜力。
已鉴定出一些干细胞和祖细胞群体，可用于心脏修复。每种细胞都具有独特的潜能，值得考虑使用。早期临床试验使用自体、未分离的骨髓细胞或骨骼肌成肌细胞来评估对近期或陈旧性梗死的修复效果。在每种情况下，都获得了心脏功能有限改善的证据。成肌细胞移植意外地与心律失常相关，从而发现了一个安全问题。患者数量少且缺乏随机对照组，无法得出关于疗效的结论。为此必须进行随机对照、中等规模、双盲临床试验。
细胞疗法可能对治疗成人心脏病有用。可能需要进行适当调整，以满足小儿心血管疾病治疗的独特要求。尽管观察到功能有所改善，但骨髓和骨骼肌成肌细胞并不能促进真正的组织再生。使用具有真正（转）分化潜能的细胞进行试验，可能会阐明这种疗法作为一种修复方式的潜力和价值。制定与病理生物学一致的靶向递送最佳策略具有至关重要的临床意义。
