Chevais A, Tarbaeva N V, Golubkina Y Y, Gadzhimuradova M M, Ivashchenko K V, Ladygina D O, Vorontsova M V, Bezlepkina O B, Melnichenko G A, Mokrysheva N G
Endocrine Research Centre.
I.M. Sechenov First Moscow State Medical University (Sechenov University).
Probl Endokrinol (Mosk). 2025 May 20;71(2):22-34. doi: 10.14341/probl13564.
Congenital adrenal hyperplasia (CAH) encompasses a group of autosomal recessive disorders characterized by defects in enzymes critical for steroidogenesis, with 21-hydroxylase deficiency due to mutations in the CYP21A2 gene being the most prevalent form.Since the introduction of replacement therapy and neonatal screening programs in the 1950s, there has been a significant increase in survival rates among newborns diagnosed with CAH. However, despite these advancements, mortality associated with this condition remains disproportionately high. Achieving optimal therapeutic compensation through medication remains a complex challenge, contributing to a range of long-term complications. These complications stem from both the underlying disease and its treatment, impacting key physiological functions, including metabolism, growth and development, cardiovascular health, and fertility. These multifaceted outcomes underscore the need for ongoing research and the refinement of therapeutic approaches to better manage this intricate condition. This article presents a series of four clinical cases of CAH characterized by the absence of sustained compensation for glucoand mineralocorticoid deficiencies. These cases were further complicated by the development of large adrenal masses and ectopic testicular adrenal rest tissue (TART), emphasizing the challenges in achieving long-term disease management.
先天性肾上腺皮质增生症(CAH)是一组常染色体隐性疾病,其特征是参与类固醇生成的关键酶存在缺陷,其中因CYP21A2基因突变导致的21-羟化酶缺乏是最常见的形式。自20世纪50年代引入替代疗法和新生儿筛查项目以来,被诊断为CAH的新生儿存活率显著提高。然而,尽管有这些进展,与该疾病相关的死亡率仍然过高。通过药物实现最佳治疗补偿仍然是一项复杂的挑战,会导致一系列长期并发症。这些并发症源于潜在疾病及其治疗,影响关键生理功能,包括代谢、生长发育、心血管健康和生育能力。这些多方面的结果凸显了持续研究和改进治疗方法以更好管理这一复杂疾病的必要性。本文介绍了一系列4例CAH临床病例,其特征是糖皮质激素和盐皮质激素缺乏未得到持续补偿。这些病例因肾上腺巨大肿块和异位睾丸肾上腺残余组织(TART)的出现而进一步复杂化,强调了实现长期疾病管理的挑战。