Statins in lymphangioleiomyomatosis: a word of caution.

Lymphangioleiomyomatosis (LAM) is a rare progressive cystic lung disease affecting primarily females, characterised by the proliferation of neoplastic LAM cells with mutations in the tuberous sclerosis complex (TSC)1 or TSC2 genes. There is no proven therapy for LAM, but results of studies with sirolimus, an inhibitor of the mammalian target of rapamycin (mTOR), have been promising [1]. Motivated by the finding [2] that in vitro, statins, which inhibit 3-hydroxy-3-methyl-glutarycoenzyme-A (HMG-CoA) reductase, inhibited the growth of TSC2 cells, we evaluated the effects of statins on the decline of lung function in 335 patients with LAM.