MANAGEMENT OF ENDOCRINE DISEASE: Regenerative therapies in autoimmune Addison's disease.
MANAGEMENT OF ENDOCRINE DISEASE: Regenerative therapies in autoimmune Addison's disease.
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DOI：
10.1530/eje-16-0581
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发表时间：
2017-03
影响因子：
5.8
通讯作者：
E. Gan;Simon H Pearce
中科院分区：
文献类型：
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作者：
E. Gan;Simon H Pearce
过去 60 年来，自身免疫性阿狄森氏病 (AAD) 的治疗方法几乎没有变化。大多数患者的症状通过外源性类固醇替代得到相对较好的控制，但尽管目前最佳治疗的依从性良好，但可能会出现持续症状、反复出现肾上腺危象和生活质量差。传统的外源性类固醇疗法还与过早死亡、心血管风险增加以及与过量类固醇替代相关的并发症有关。因此，在过去十年中出现了新的治疗方法，试图改善 AAD 患者的长期结果和生活质量。这篇综述讨论了 AAD 治疗创新的最新进展，包括旨在模仿皮质醇分泌的生理生物节律的新型外源类固醇制剂。我们的小组还进行了一些研究，试图通过免疫调节和再生医学方法恢复内源性糖皮质激素的产生。还将讨论对肾上腺皮质干细胞生物学和肾上腺可塑性的理解的最新进展，以帮助理解所采用的治疗方法背后的科学原理。
The treatment for autoimmune Addison's disease (AAD) has remained virtually unchanged in the last 60 years. Most patients have symptoms that are relatively well controlled with exogenous steroid replacement, but there may be persistent symptoms, recurrent adrenal crisis and poor quality of life, despite good compliance with optimal current treatments. Treatment with conventional exogenous steroid therapy is also associated with premature mortality, increased cardiovascular risk and complications related to excessive steroid replacement. Hence, novel therapeutic approaches have emerged in the last decade attempting to improve the long-term outcome and quality of life of patients with AAD. This review discusses the recent developments in treatment innovations for AAD, including the novel exogenous steroid formulations with the intention of mimicking the physiological biorhythm of cortisol secretion. Our group has also carried out a few studies attempting to restore endogenous glucocorticoid production via immunomodulatory and regenerative medicine approaches. The recent advances in the understanding of adrenocortical stem cell biology, and adrenal plasticity will also be discussed to help comprehend the science behind the therapeutic approaches adopted.