Novel treatment strategies in congenital adrenal hyperplasia

In recent years, important steps have been taken to improve the treatment of congenital adrenal hyperplasia (CAH), a relatively stagnant area for decades. In this review, we summarize these advances and propose future lines of investigation.

The two main goals of CAH treatment are to replace the deficient hormones when necessary and to dampen the adrenocorticotropin activation and the ensuing adrenal androgen excess. Glucocorticoids have been the mainstay of CAH treatment, but available preparations only partially meet the clinical needs. Recent efforts have focused on improving the delivery of glucocorticoid replacement agents, to closer mimic the physiologic secretion pattern. Examples include modified release oral glucocorticoids and continuous subcutaneous hydrocortisone pumps. Furthermore, nonglucocorticoid approaches to address the androgen excess have emerged, such as inhibition of key androgenic enzymes and adrenocorticotropin secretion blockade by corticotropin-releasing hormone receptor antagonists.

The promising recent progress made in CAH treatment brings new perspectives for individualized care in this complex disease.