New ideas for medical treatment of congenital adrenal hyperplasia

Since the introduction of glucocorticoid and mineralocorticoid replacement therapy approximately 50 years ago, the standard treatment principles of 21-hydroxylase deficiency have remained largely unchanged. Glucocorticoid is given in doses sufficient to suppress adrenal androgen secretion, and mineralocorticoid is given to normalize electrolytes and plasma renin activity. This treatment strategy often fails to normalize the growth and development of children with congenital adrenal hyperplasia (CAH), and management is often complicated by iatrogenic Cushing's syndrome, inadequately treated hyperandrogenism, and, later in life, infertility. This article addresses the clinical problems patients with CAH continue to have and outlines new ideas for the medical treatment of this condition.