CRH受体拮抗剂crinecerfont--21-羟化酶缺乏症所致先天性肾上腺皮质增生症患者的一种有希望的新治疗方案。

IF 1.3 4区 医学 Q4 ENDOCRINOLOGY & METABOLISM Journal of Pediatric Endocrinology & Metabolism Pub Date : 2024-11-25 DOI:10.1515/jpem-2024-0440
Clemens Kamrath, Hedi L Claahsen-van der Grinten
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引用次数: 0

摘要

21-羟化酶缺乏症(21OHD)是先天性肾上腺皮质增生症(CAH)中最常见的一种,会导致皮质醇合成障碍和雄激素过多。目前使用超生理剂量的糖皮质激素治疗典型的 21OHD 患者会带来生长受阻和代谢并发症等风险。我们讨论了 CRH 受体拮抗剂作为治疗典型 21OHD 儿童的一种选择。CRH受体拮抗剂Crinecerfont的三期试验提供了一种很有前景的新治疗方案。Crinecerfont有助于减少糖皮质激素剂量,降低雄激素水平。然而,这项研究的人群可能并不能完全代表 21OHD 的一般人群。能否成功实施取决于患者的依从性和监测,以避免可能出现的并发症,如肾上腺危象。总的来说,crinecerfont是一项有价值的研发成果,但要优化其在CAH治疗中的应用,还需要进一步的研究和谨慎的临床管理。
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CRH receptor antagonist crinecerfont - a promising new treatment option for patients with congenital adrenal hyperplasia due to 21-hydroxylase deficiency.

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.

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来源期刊
CiteScore
2.70
自引率
7.10%
发文量
176
审稿时长
3-6 weeks
期刊介绍: The aim of the Journal of Pediatric Endocrinology and Metabolism (JPEM) is to diffuse speedily new medical information by publishing clinical investigations in pediatric endocrinology and basic research from all over the world. JPEM is the only international journal dedicated exclusively to endocrinology in the neonatal, pediatric and adolescent age groups. JPEM is a high-quality journal dedicated to pediatric endocrinology in its broadest sense, which is needed at this time of rapid expansion of the field of endocrinology. JPEM publishes Reviews, Original Research, Case Reports, Short Communications and Letters to the Editor (including comments on published papers),. JPEM publishes supplements of proceedings and abstracts of pediatric endocrinology and diabetes society meetings.
期刊最新文献
Age and sex mark clinical differences in the presentation of pediatric type 1 diabetes mellitus. Geographic information system mapping and predictors of glycemic control in children and youth with type 1 diabetes: a study from Western India. Persistent hypoglycemia in congenital syphilis: hyperinsulinemic hypoglycemia with a focal pancreatic lesion. Assessment of quality of life in families affected by maple syrup urine disease: a cross sectional study. CRH receptor antagonist crinecerfont - a promising new treatment option for patients with congenital adrenal hyperplasia due to 21-hydroxylase deficiency.
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