This protocol addresses classic congenital adrenal hyperplasia in adults aged 18 years and older in whom hyperandrogenic symptoms remain inadequately controlled despite established glucocorticoid and mineralocorticoid replacement therapy.
Adults with classic CAH require ongoing glucocorticoid and mineralocorticoid replacement. In young women, however, hyperandrogenic symptoms may persist even when the foundational hormonal regimen is in place, pointing to the need for an additional treatment step.
Standard first-line management of classic CAH in adults uses glucocorticoid replacement — hydrocortisone or a long-acting glucocorticoid (prednisone, prednisolone, methylprednisolone, or dexamethasone) — together with mineralocorticoid replacement with fludrocortisone. The targets for that regimen include androstenedione and testosterone at or near normal levels, and, for women trying to conceive, an appropriate follicular-phase progesterone level. When hyperandrogenic symptoms in young women remain uncontrolled despite this regimen, a further step is indicated — which is the focus of this protocol.
In young women with classic CAH whose hyperandrogenic symptoms persist on glucocorticoid and mineralocorticoid therapy, the evidence-based approach involves adding a targeted agent directed at androgen excess alongside the existing regimen. The complete protocol — including which agent, sequencing, and all clinical criteria — is available below.
DOI: 10.1210/jc.2018-01865