Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis.

ABSTRACT:

Background

Angiomyolipomas in patients with the tuberous sclerosis complex or sporadic lymphangioleiomyomatosis are associated with mutations in tuberous sclerosis genes resulting in constitutive activation of the mammalian target of rapamycin (mTOR). The drug sirolimus suppresses mTOR signaling.

Methods

We conducted a 24-month, nonrandomized, open-label trial to determine whether sirolimus reduces the angiomyolipoma volume in patients with the tuberous sclerosis complex or sporadic lymphangioleiomyomatosis. Sirolimus was administered for the first 12 months only. Serial magnetic resonance imaging of angiomyolipomas and brain lesions, computed tomography of lung cysts, and pulmonary-function tests were performed.

Results

Of the 25 patients enrolled, 20 completed the 1

SUBMITTER: Bissler JJ 

PROVIDER: S-EPMC3398441 | biostudies-literature | 2008 Jan

REPOSITORIES: biostudies-literature