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2016 ; 23
(3
): 225-32
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Novel treatment strategies in congenital adrenal hyperplasia
#MMPMID27032061
Turcu AF
; Auchus RJ
Curr Opin Endocrinol Diabetes Obes
2016[Jun]; 23
(3
): 225-32
PMID27032061
show ga
PURPOSE OF REVIEW: 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. RECENT FINDINGS: 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. SUMMARY: The promising
recent progress made in CAH treatment brings new perspectives for individualized
care in this complex disease.