Efficacy and safety of sirolimus in lymphangioleiomyomatosis.

ABSTRACT:

Background

Lymphangioleiomyomatosis (LAM) is a progressive, cystic lung disease in women; it is associated with inappropriate activation of mammalian target of rapamycin (mTOR) signaling, which regulates cellular growth and lymphangiogenesis. Sirolimus (also called rapamycin) inhibits mTOR and has shown promise in phase 1-2 trials involving patients with LAM.

Methods

We conducted a two-stage trial of sirolimus involving 89 patients with LAM who had moderate lung impairment--a 12-month randomized, double-blind comparison of sirolimus with placebo, followed by a 12-month observation period. The primary end point was the difference between the groups in the rate of change (slope) in forced expiratory volume in 1 second (FEV(1)).

Results

During the treatment period, the F

SUBMITTER: McCormack FX 

PROVIDER: S-EPMC3118601 | biostudies-literature | 2011 Apr

REPOSITORIES: biostudies-literature