LAUSR

Introduction: Lymphangioleiomiomatosis (LAM) is a rare, progressive cystic lung disease, mainly affecting women. Sirolimus (SR) is effective in stabilizing lung function and in reducing the size of chylous effusions, lymphangioleiomyomas, and angiomyolipomas Objectives: Aim of this study is to analyze data of patients (pts) referred to our LAM clinic and treated with SR Methods: We retrospectively analyzed data of pts evaluated after at least one year of treatment with pulmonary function tests (PFTs) and six minute walking test (6MWT) Results: 63 LAM pts, 16 associated to tuberous sclerosis (TSC), were treated with SR since 2009. 13 discontinued: 5 for disease progression, 2 for adverse events (SE), 1 died and 5 lost at follow-up. 39 and 31 were respectively evaluated at 12 and 24 months. No significant changes were observed in FVC, FEV1, DLCO and 6MWT at 12 and 24 months compared to baseline. However 5 pts (non responders) showed a decrease of ≥10% in FEV1 after 12 month treatments, confirmed at the evaluation at 24 m. These pts did not show any significant difference in PFTs and 6MWD at baseline compared to the latters. Among responders, a significant correlation was found between FEV1 at baseline and FEV 1 at 12 (rho 0.9, p In 26 pts also PFTs before treatment were available. In this subgroup post treatment-changes in FEV1 at 12 months and for 21 patients at 24 months were significantly different from yearly change before treatment (-0.1 vs 0.04 p=0.03 and -0.1 vs 0.02 L/year p =0.05 respectively). Conclusions: Our data confirm the important role played by SR in slowing down LAM progression. However around 20% of pts experienced diseases progression or AE.