Nambam Bimota, Haller Michael J
Division of Endocrinology, Louisiana State University, Shreveport, US.
Division of Endocrinology, University of Florida, Gainesville, US.
Eur Endocrinol. 2016 Aug;12(2):89-95. doi: 10.17925/EE.2016.12.02.89. Epub 2016 Aug 28.
Multiple clinical trials investigating the efficacy and safety of immunotherapeutic interventions in new onset type 1 diabetes (T1D) have failed to yield long term clinical benefit. Lack of efficacy has frequently been attributed to an incomplete understanding of the pathways involved in T1D and the use of single immunotherapeutic agents. Recent mechanistic studies have improved our knowledge of the complex etiopathogenesis of T1D. This in turn has provided the framework for new and ongoing clinical trials in new onset T1D patients and at-risk subjects. Focus has also shifted towards the potential benefits of synergistic combinatorial approaches, both in terms of efficacy and the potential for reduced side effects. These efforts seek to develop intervention strategies that will preserve β-cell function, and ultimately prevent and reverse clinical disease.
多项针对新发性1型糖尿病(T1D)免疫治疗干预措施的疗效和安全性的临床试验均未产生长期临床益处。疗效不佳常常归因于对T1D所涉及途径的理解不完整以及单一免疫治疗药物的使用。最近的机制研究增进了我们对T1D复杂病因发病机制的了解。这反过来为针对新发性T1D患者和高危受试者开展新的和正在进行的临床试验提供了框架。关注点也已转向协同组合方法在疗效和降低副作用可能性方面的潜在益处。这些努力旨在制定能够保留β细胞功能并最终预防和逆转临床疾病的干预策略。