Stem cell therapy for CHD: towards translation.
Stem cell therapy for CHD: towards translation.
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冠心病的干细胞疗法：走向转化。
DOI：
10.1017/s1047951115000840
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发表时间：
2015
影响因子：
1
通讯作者：
Kaushal,Sunjay
中科院分区：
文献类型：
--
作者：
Wehman,Brody;Siddiqui,OsamaT;Mishra,Rachana;Sharma,Sudhish;Kaushal,Sunjay
干细胞治疗的乐观目标是使心肌再生，定义为使丢失或破坏的心肌再生。当应用于心力衰竭患者时，许多人混淆或限制再生的定义仅仅是改善心肌功能和/或减少心肌瘢痕形成，这可能不是分子医学这个有前途的领域最重要的临床结果。许多不同的基于干细胞的治疗方法已经被测试，并证明在成年心力衰竭患者中是安全可行的，但报道的有效终点不同。虽然目前还没有实现，但是在成年心力衰竭患者中使用这些基于细胞的治疗方法来再生心脏的目标仍然被认为是可以实现的，因为第一代治疗方法正在临床试验的不同阶段进行测试。然而，促进将干细胞疗法转化为心力衰竭儿童的类似努力受到限制。在这篇综述中，我们的目的是总结临床前模型和临床经验的发现，迄今为止，这些研究都集中在对心力衰竭儿童干细胞治疗的评估上。最后，我们提出了与心脏衰竭儿童干细胞试验设计相关的方法学考虑，因为与成人患者相比，他们代表了具有非常不同问题的患者群体。正如许多博学的临床医生所教导的那样，儿童不是小大人！
Stem cell therapy has the optimistic goal of regenerating the myocardium as defined by re-growth of lost or destroyed myocardium. As applied to patients with heart failure, many confuse or limit the regenerative definition to just improving myocardial function and/or decreasing myocardial scar formation, which may not be the most important clinical outcome to achieve in this promising field of molecular medicine. Many different stem cell-based therapies have been tested and have demonstrated a safe and feasible profile in adult patients with heart failure, but with varied efficacious end points reported. Although not achieved as of yet, the encompassing goal to regenerate the heart is still believed to be within reach using these cell-based therapies in adult patients with heart failure, as the first-generation therapies are now being tested in different phases of clinical trials. Similar efforts to foster the translation of stem cell therapy to children with heart failure have, however, been limited. In this review, we aim to summarise the findings from pre-clinical models and clinical experiences to date that have focussed on the evaluation of stem cell therapy in children with heart failure. Finally, we present methodological considerations pertinent to the design of a stem cell-based trial for children with heart failure, as they represent a population of patients with very different sets of issues when compared with adult patients. As has been taught by many learned clinicians, children are not small adults!