{"title":"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。","authors":"Clemens Kamrath, Hedi L Claahsen-van der Grinten","doi":"10.1515/jpem-2024-0440","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":" ","pages":""},"PeriodicalIF":1.3000,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"CRH receptor antagonist crinecerfont - a promising new treatment option for patients with congenital adrenal hyperplasia due to 21-hydroxylase deficiency.\",\"authors\":\"Clemens Kamrath, Hedi L Claahsen-van der Grinten\",\"doi\":\"10.1515/jpem-2024-0440\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":50096,\"journal\":{\"name\":\"Journal of Pediatric Endocrinology & Metabolism\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":1.3000,\"publicationDate\":\"2024-11-25\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Pediatric Endocrinology & Metabolism\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1515/jpem-2024-0440\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"ENDOCRINOLOGY & METABOLISM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Pediatric Endocrinology & Metabolism","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1515/jpem-2024-0440","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}
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.
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.