Lymphangioleiomyomatosis treatment with sirolimus

Lymphangioleiomyomatosis (LAM) is a rare lung disease, that predominantly affects young females and generally progresses to respiratory failure. There is not sufficient evidence to support the routine use of any treatment in LAM. The only treatment for severe LAM is currently lung transplantation. Activation of mammalian target of rapamycin (mTOR) signalling pathway has been observed in LAM. LAM is often associated with angiomyolipoma in the kidneys. mTOR inhibitor sirolimus reduces angymiolipoma volumes. Some reports have shown improvement in lung function with sirolimus in LAM. We report 3 women with LAM, with a rapid decline in lung function and symptoms and who were treated with sirolimus.