Mifepristone Treatment in Four Cases of Primary Bilateral Macronodular Adrenal Hyperplasia (BMAH).

CONTEXT: Primary bilateral macronodular adrenal hyperplasia (BMAH) is a rare form of adrenal Cushing syndrome conventionally treated with adrenalectomy. Medical treatment is often reserved for patients not eligible for surgery. However, to date, there have been few studies about the efficacy of mifepristone for the treatment of BMAH associated with hypercortisolism. OBJECTIVE: To describe a series of patients with hypercortisolism due to BMAH treated with mifepristone. DESIGN: We retrospectively assessed 4 patients treated with mifepristone from multiple medical practices with hypercortisolism due to BMAH had who either failed unilateral adrenalectomy, declined surgery, or were poor surgical candidates. RESULTS: Mifepristone induced clinical improvement and remission of the signs and symptoms of hypercortisolism in all the described patients with BMAH. The median treatment duration at the time of efficacy response assessment was 5 months (range: 3-18 months). Improvement in cardiometabolic parameters was observed as early as 2 weeks after treatment was started. All patients achieved improvements in glycemic control and hypertension and had significant weight loss. The most common adverse event observed with mifepristone therapy was fatigue. Increases in thyroid-stimulating hormone level occurred in 2 patients. CONCLUSION: Mifepristone can be an effective alternative to surgery in patients with hypercortisolism due to BMAH