Renal angiomyolipoma and lymphangiomleiomyoma sirolimus treatment in patients with lymphangioleiomyomatosis.

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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. Material and methods: 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. Results: 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. Conclusions: 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.

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