Lung function response and side effects to rapamycin for lymphangioleiomyomatosis: a prospective national cohort study

Rationale Mechanistic target of rapamycin inhibitors reduce loss of lung function in lymphangioleiomyomatosis (LAM), although their benefit varies between individuals. We examined lung function response and side effects to rapamycin in a national cohort. Methods Subjects were receiving rapamycin for progressive lung disease. Clinical evaluation, detailed phenotyping, serial lung function, rapamycin and safety monitoring were performed according to a clinical protocol. Lung function change, measured as FEV1 slope (ΔFEV1), was reported for those treated for 1 year or longer. Results Rapamycin was associated with improved ΔFEV1 in 21 individuals where pretreatment data were available (p