Cabergoline for Cushing's disease: a large retrospective multicenter study.

Area of Science:

• Endocrinology
• Pharmacology

Background:

• Cushing's disease (CD) is a rare endocrine disorder caused by excess cortisol production.
• The efficacy of cabergoline, a dopamine agonist, in treating CD remains debated.

Purpose of the Study:

• To evaluate the efficacy and tolerability of cabergoline in a large cohort of patients with Cushing's disease.
• To identify predictors of response to cabergoline therapy in CD.

Main Methods:

• A retrospective multicenter study involving 62 patients with CD across 13 university hospitals.
• Data collected included symptom scores, biological markers of hypercortisolism (urinary free cortisol - UFC, midnight cortisol, plasma ACTH), and adverse effects.
• Patients received either cabergoline monotherapy or add-on therapy to existing treatments.

Main Results:

• 40% of patients on monotherapy achieved normal UFC within 12 months (complete responders).
• Complete responders showed significant reductions in hypercortisolism markers and clinical improvement, often at lower cabergoline doses.
• Sustained hypercortisolism control was achieved in 23% of patients long-term; 56% of patients receiving cabergoline as add-on therapy also showed control.

Conclusions:

• Approximately 20-25% of Cushing's disease patients are good responders to cabergoline, achieving long-term hypercortisolism control.
• Cabergoline demonstrated acceptable tolerability, but treatment escape or intolerance led to discontinuation in 28% of responders.
• No specific baseline parameters, such as UFC or prolactin levels, could predict response to cabergoline therapy.