Novel treatments for congenital adrenal hyperplasia

Schroder Mariska A. M.; Claahsen-van der Grinten Hedi L.

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Novel treatments for congenital adrenal hyperplasia

机译：Novel treatments for congenital adrenal hyperplasia

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Patients with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (21OHD) need life-long medical treatment to replace the lacking glucocorticoids and potentially lacking mineralocorticoids and to lower elevated adrenal androgens. Long-term complications are common, including gonadal dysfunction, infertility, and cardiovascular and metabolic co-morbidity with reduced quality of life. These complications can be attributed to the exposure of supraphysiological dosages of glucocorticoids and the longstanding exposure to elevated adrenal androgens. Development of novel therapies is necessary to address the chronic glucocorticoid overexposure, lack of circadian rhythm in glucocorticoid replacement, and inefficient glucocorticoid delivery with concomitant periods of hyperandrogenism. In this review we aim to give an overview about the current treatment regimens and its limitations and describe novel therapies especially evaluated for 21OHD patients.

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《Reviews in endocrine & metabolic disorders》 |2022年第3期|631-645|共15页
作者
Schroder Mariska A. M.; Claahsen-van der Grinten Hedi L.;
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作者单位

Amalia Childrens Hosp,Radboud Univ Nijmegen;

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正文语种英语
中图分类内分泌腺疾病及代谢病;
关键词
Congenital Adrenal Hyperplasia; 21-hydroxylase deficiency; Glucocorticoid replacement therapy; Modified-release; Corticotropin-releasing hormone receptor antagonist; Adrenocorticotropic hormone antagonist; Steroid production inhibitors; SUBCUTANEOUS HYDROCORTISONE INFUSION; MODIFIED-RELEASE HYDROCORTISONE; 21-HYDROXYLASE DEFICIENCY; ORAL HYDROCORTISONE; ADRENOCORTICAL-CELLS; REPLACEMENT THERAPY; ABIRATERONE ACETATE; ADDISONS-DISEASE; GROWTH VELOCITY; INSUFFICIENCY;

Novel treatments for congenital adrenal hyperplasia

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