Renal angiomyolipoma and lymphangiomleiomyoma sirolimus treatment in patients with lymphangioleiomyomatosis.

Lymphangioleiomyomatosis (LAM) is a rare disease caused by uncontrolled proliferation of cells bearing the mutation of TSC1 and/or TSC2 genes. Polycystic lung destruction, chylous pleural or abdominal effusions, lymphangioleiomyomas (LYMPH), angiomyolipoma’s (AML) are noticed in the course of the disease. MTOR inhibitors have been approved for LAM treatment. <bold>Material and methods:</bold> In the period of 2010-2020, 71 patients with LAM (age ranging; 24 - 76 years), were treated with sirolimus. AML’s were detected in 35 out of 71 patients and LYMPH in 27 patients with LAM. <bold>Results:</bold> There was a significant reduction in the mean volume of renal AML after 3 months (80 ± 51 vs. 103 ± 106mm³; p=0.002), 1 year (93 ± 100 vs. 103 ± 106mm³; p<0.000), 2 years (88 ± 69 vs. 103 ± 106mm³; p<0.000), and 5 years of therapy (49 ± 44 vs. 103 ± 106mm³; p=0.008). Gradual decrease of the mean volume of LYMPH was noticed; after 3 months of therapy (193 ± 144 vs. to 113 ± 141mm³; p<0.000), after 1 year to 91 ± 143mm³; p<0.000, after 2 years to 81 ± 137mm³; p<0.000 and after 5 to 74 ± 139mm³; p=0.001. After 5 years of treatment a 52% reduction in AML volume, and a 62% reduction in LYMPH volume were observed. <bold>Conclusions:</bold> Sirolimus treatment resulted in significant regression of AML and LYMPH volume, The long-term sirolimus treatment is safe in patients with LAM, and its effects were maintained during long-term follow-up.