Yoo Han-Wook
Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
Ann Pediatr Endocrinol Metab. 2021 Sep;26(3):149-157. doi: 10.6065/apem.2142150.075. Epub 2021 Sep 30.
Primary adrenal insufficiency (PAI) in pediatric age is a rare, but potentially fatal condition caused by diverse etiologies including biochemical defects of steroid biosynthesis, developmental abnormalities of the adrenal gland, or reduced responsiveness to adrenocorticotropic hormone. Compared to adult PAI, pediatric PAI is more often the result of genetic (monogenic, syndromic disorders) than acquired conditions. During the past decade, rare monogenic disorders associated with PAI have helped unravel the underlying novel molecular genetic mechanism. The diagnosis of adrenal insufficiency in children and young infancy is often challenging, usually based on clinical suspicion and endocrine laboratory findings. Pediatric endocrinologists sometimes encounter therapeutic difficulty in finding the balance between undertreatment and overtreatment, determining how to optimize the dose over the patient's lifetime, and maximizing mimicry of normal cortisol secretion with glucocorticoid replacement therapy.
儿童期原发性肾上腺皮质功能减退症(PAI)较为罕见,但可能致命,其病因多样,包括类固醇生物合成的生化缺陷、肾上腺发育异常或对促肾上腺皮质激素反应性降低。与成人PAI相比,儿童PAI更常由遗传因素(单基因、综合征性疾病)而非后天性疾病导致。在过去十年中,与PAI相关的罕见单基因疾病有助于揭示潜在的新分子遗传机制。儿童及幼儿期肾上腺皮质功能减退症的诊断往往具有挑战性,通常基于临床怀疑和内分泌实验室检查结果。儿科内分泌学家有时在治疗中会遇到困难,即在治疗不足与过度治疗之间找到平衡,确定如何在患者一生中优化剂量,以及通过糖皮质激素替代疗法最大程度地模拟正常皮质醇分泌。