Kamrath Clemens, Claahsen-van der Grinten Hedi L
Division of Pediatric Endocrinology and Diabetology, Centre for Paediatrics and Adolescent Medicine, 88751 University of Freiburg , Freiburg, Germany.
Department of Pediatrics, Division of Pediatric Endocrinology, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands.
J Pediatr Endocrinol Metab. 2024 Nov 25;38(1):16-21. doi: 10.1515/jpem-2024-0440. Print 2025 Jan 29.
21-Hydroxylase deficiency (21OHD), the most common form of congenital adrenal hyperplasia (CAH), leads to impaired cortisol synthesis and androgen excess. Current treatments of patients with classic 21OHD with supraphysiological doses of glucocorticoids pose risks such as impaired growth and metabolic complications. We discuss the CRH receptor antagonist as a therapeutic option for children with classic 21OHD. A phase three trial of crinecerfont, a CRH receptor antagonist, offers a promising new treatment option. Crinecerfont helped to reduce glucocorticoid doses and to lower androgen levels. However, the study population may not be fully representative of the general 21OHD population. Successful implementation depends on patient adherence and monitoring to avoid possible complications such as adrenal crises. Overall, crinecerfont represents a valuable development, but further research and careful clinical management are needed to optimize its use in CAH treatment.
21-羟化酶缺乏症(21OHD)是先天性肾上腺皮质增生症(CAH)最常见的形式,会导致皮质醇合成受损和雄激素过量。目前,使用超生理剂量糖皮质激素治疗典型21OHD患者存在生长受损和代谢并发症等风险。我们讨论了促肾上腺皮质激素释放激素(CRH)受体拮抗剂作为典型21OHD儿童的一种治疗选择。CRH受体拮抗剂可宁塞丰的三期试验提供了一种有前景的新治疗选择。可宁塞丰有助于减少糖皮质激素剂量并降低雄激素水平。然而,研究人群可能无法完全代表一般21OHD人群。成功应用取决于患者的依从性和监测,以避免诸如肾上腺危象等可能的并发症。总体而言,可宁塞丰是一项有价值的进展,但需要进一步研究和谨慎的临床管理,以优化其在CAH治疗中的应用。