This protocol covers the clinical scenario in which a child under 18 years of age with Cushing's disease has undergone first-line surgical treatment, but adrenal function has not recovered within the expected timeframe. It defines the next management step for this paediatric population.
Endogenous Cushing's syndrome is extraordinarily rare before age 18. In children over age 6, Cushing's disease is the most common cause of endogenous Cushing's syndrome, while adrenal causes are more common in younger children.
Surgical resection of the ACTH-secreting tumour via transsphenoidal surgery is the established first-line treatment. This protocol is indicated when adrenal function has not recovered within approximately 12 months following that surgery.
Medical therapy is the next consideration, centred on agents that reduce cortisol production. Monitoring of morning cortisol levels is used to guide the therapeutic response. An alternative regimen approach may also be considered. The complete protocol — including which agents, sequencing, and full monitoring guidance — is available via the structured regimen below.
DOI: 10.1016/S2213-8587(21)00235-7
Endogenous CS is extraordinarily rare before age 18.
In children over age 6, CD is the most common cause of CS, while adrenal causes are more common in younger children.
For those requiring medical therapy, ketoconazole or metyrapone is typically used with morning cortisol for monitoring response.
Pasireotide is not recommended and clinical trials of osilodrostat in children are underway.
Block-and-replace regimens with metyrapone also may be considered.
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