Treatment patterns in Cushing's disease patients in two large United States nationwide databases: application of a...

Data on real-world treatment patterns for Cushing's disease (CD) are limited. We used a novel graphical technique to analyze treatment patterns in CD patients in the United States.

Two combined US claims databases were used to identify CD patients with claims with Cushing's syndrome diagnosis and either benign pituitary adenoma or hypophysectomy and newly-treated in 2008 (no treatment in prior 6 months). Patients were followed from first treatment day until end of enrollment or 12/31/2010. We compared summary statistics with a novel graphical methodology that simultaneously displays individual color-coded patient treatment histories.

Among 228 newly-treated CD patients, 180 (78.9%) had surgery as first observed treatment, 42 (18.4%) had pharmacotherapy, and 6 (2.6%) had radiotherapy. In 42 patients who had pharmacotherapy as first treatment, dopamine agonists were used as first pharmacotherapy in 24 (57.1%), ketoconazole in 17 (40.5%), and mitotane in one patient (2.4%). In 180 patients with surgery as first treatment, 15 (8.3%) later had radiotherapy and 14 (7.8%) had pharmacotherapy. In 42 patients who had pharmacotherapy as first treatment, 10 (23.8%) later had surgery and 2 (4.8%) had radiotherapy. Mean duration of first pharmacotherapy varied: 369.5 days for dopamine agonists, 157.1 for ketoconazole, and 30.0 for mitotane.

This study addresses a need for US data on real-world treatment patterns for CD patients. The majority of CD patients undergo surgery as initial therapy. Patients using pharmacotherapy had limited persistence with treatment. Neither reasons for discontinuation of therapy nor the impact of a recent FDA warning on potentially fatal liver toxicity from ketoconazole could be assessed.