Emerging treatments
Novel hydrocortisone formulations
Hydrocortisone microgranules (or sprinkles) are available in some countries as an alternative to the standard formulation of hydrocortisone tablets. They are available in various strengths, thus allowing for small dose titrations, which is especially important for young children. The granule/sprinkle formulation is available in the US and Europe. A modified-release formulation of hydrocortisone is also available in some countries as an alternative to the standard formulation of hydrocortisone tablets. The modified-release formulation was specifically designed for patients with CAH and allows for the slow release of hydrocortisone during the night with the aim of suppressing the rise of adrenocorticotropic hormone (ACTH) and adrenal androgens in early morning hours. The modified-release formulation is available in Europe, but not the US as yet.
Crinecerfont
Crinecerfont, a corticotropin-releasing factor type 1 (CRFR1) receptor antagonist is currently undergoing trials as an adjunct therapy for patients with CAH. The drug suppresses ACTH signaling independent of glucocorticoid replacement, and thus, improves adrenal hyperandrogenism. Preliminary results are promising.[30]
Prenatal dexamethasone
Prenatal low-dose dexamethasone treatment is effective in preventing virilization in 46,XX individuals. However, the treatment is controversial because of concerns that prenatal dexamethasone exposure may lead to long-term adverse effects in adulthood from neurodevelopmental disorders to impaired beta cell function.[31] Guidelines from the Endocrine Society consider prenatal low-dose dexamethasone therapy to be experimental and do not endorse specific treatment protocols. They also recommend that patients only obtain prenatal therapy through protocols approved by Institutional Review Boards at centers capable of collecting outcomes from a sufficiently large number of patients.[14]
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