Division of Endocrinology, Department of Pediatrics, University of Florida, PO Box 100296, Gainesville, FL 32610, USA.
Endocrinol Metab Clin North Am. 2010 Sep;39(3):527-39. doi: 10.1016/j.ecl.2010.05.006.
Despite improvements in understanding of the natural history of type 1 diabetes (T1D), an intervention capable of consistently and safely preventing or reversing the disease has not been developed. The inability to cure this disorder is largely because of the complex pathophysiology of T1D, continued struggles to identify its precise etiologic triggers, and voids in understanding of the immunologic mechanisms that specifically target pancreatic beta cells. Rapidly improving technologies for managing T1D require critical discussions about equipoise, especially when considering interventions deemed high risk in terms of their safety. This article reviews the conceptual basis for prevention versus intervention trials in settings of T1D, past experiences of clinical trials studying these purposes, and controversial issues regarding disease interdiction, and seeks to provide a roadmap for future efforts to cure this disorder.
尽管人们对 1 型糖尿病(T1D)的自然史有了更多的了解,但仍未能开发出一种能够持续、安全地预防或逆转该疾病的干预措施。之所以无法治愈这种疾病,主要是因为 T1D 的病理生理学非常复杂,难以确定其确切的病因触发因素,也无法理解针对胰腺β细胞的免疫机制。在管理 T1D 的技术不断快速改进的情况下,需要就平衡状态进行关键性讨论,尤其是在考虑被认为存在安全风险的干预措施时。本文回顾了在 T1D 环境下预防与干预试验的概念基础、过去研究这些目的的临床试验经验,以及关于疾病干预的有争议问题,并试图为治愈该疾病的未来努力提供路线图。