Kılınç Uğurlu Aylin, Özsu Elif, Aycan Zehra
Gazi University Faculty of Medicine, Department of Pediatric Endocrinology, Ankara, Turkey
Ankara University Faculty of Medicine, Department of Pediatric Endocrinology, Ankara, Turkey
J Clin Res Pediatr Endocrinol. 2025 Jan 10;17(Suppl 1):60-65. doi: 10.4274/jcrpe.galenos.2024.2024-6-22-S. Epub 2024 Dec 23.
Congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency accounts for approximately 95% of all CAH cases and is one of the most common inborn errors of metabolism. While glucocorticoid therapy has significantly improved patient outcomes, the focus has shifted towards managing the long-term effects. Numerous adverse outcomes have been associated with CAH, including those resulting from supraphysiological doses of glucocorticoid and mineralocorticoid replacement, excessive adrenal androgen secretion, and elevated levels of steroid precursors and adrenocorticotropic hormone. Despite advances in treatment, long-term complications persist due to the inability to replicate physiological hormone secretion fully. In this review, we explore critical aspects of managing CAH, focusing on cardiometabolic health, bone integrity, fertility, and other significant long-term consequences, informed by the latest literature.
由于21-羟化酶缺乏导致的先天性肾上腺皮质增生症(CAH)约占所有CAH病例的95%,是最常见的先天性代谢缺陷之一。虽然糖皮质激素治疗显著改善了患者的预后,但重点已转向管理长期影响。CAH与许多不良后果相关,包括超生理剂量的糖皮质激素和盐皮质激素替代、肾上腺雄激素分泌过多以及类固醇前体和促肾上腺皮质激素水平升高所导致的后果。尽管治疗取得了进展,但由于无法完全复制生理性激素分泌,长期并发症仍然存在。在本综述中,我们根据最新文献探讨了CAH管理的关键方面,重点关注心脏代谢健康、骨骼完整性、生育能力以及其他重大的长期后果。