Rationale: Lymphangioleiomyomatosis (LAM) is a progressive cystic lung disease that predominantly affects women and can worsen with pregnancy, estrogen treatment, and the menstrual cycle, suggesting an important role for estrogen in disease pathogenesis.
Objectives: To assess the efficacy and safety of the aromatase inhibitor letrozole in the treatment of LAM.
Methods: Seventeen postmenopausal women with LAM were enrolled in this phase II trial and randomized to receive letrozole 2.