This protocol applies to patients with Cushing's disease (CD) in whom intensive multi-drug combination medical therapy — used to maximally suppress cortisol production — did not achieve normalization of urinary free cortisol (UFC). With medical options exhausted, a different category of intervention becomes relevant.
Combination medical therapy — including ketoconazole plus metyrapone, ketoconazole plus osilodrostat, ketoconazole plus cabergoline, ketoconazole plus pasireotide, pasireotide plus cabergoline, or triplet regimens such as cabergoline plus pasireotide plus ketoconazole, or ketoconazole plus metyrapone plus mitotane — failed to normalize urinary free cortisol (UFC), triggering escalation to this protocol.
When all medical options have been exhausted, a definitive surgical intervention targeting the adrenal glands is available. It is considered a treatment of last resort, reserved for select patients, and can provide immediate control of cortisol excess where prior therapies have failed.
DOI: 10.1016/S2213-8587(21)00235-7
BLA offers immediate control of cortisol excess in patients with persistent or recurrent CD not responsive to medical therapy, but is only considered for select patients due to the resultant AI and need for life-long GC and mineralocorticoid replacement therapy.
In patients with CD, BLA is often considered a treatment of last resort in most centers after all other options have failed.
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