Elżbieta Radzikowska, Katarzyna Błasińska-Przerwa, Paulina Skrońska, Elżbieta Wiatr, Tomasz Świtaj, Agnieszka Skoczylas and Kazimierz Roszkowski-Śliż
Objective: Lymphangioleiomyomatosis (LAM) is a rare disease caused by abnormal proliferation of smooth muscle-like cells mainly in lungs and axial lymph nodes, that belongs to the group of PEComa tumours. Chylous pleural and abdominal effusions, lymhangioleiomiomas, angiomyolipomas are noticed in disease course. Sirolimus has been approved for LAM treatment, and also it has been reported to decrease the size of angiomyolipomas, lymphangiooedma, pleural and peritoneal chylous effusion. The aim of the study was to assess the significance of sirolimus therapy in patients with LAM and lymphangioma in retroperitoneal space. Method: Fourteen women with confirmed diagnosis of LAM (13 with sporadic LAM, and one TSC/LAM) and presence of lymphangioma in abdominal cavity were retrospectively reviewed. Seven patients displayed chyloperitoneum and chylothorax. Mean time of sirolimus exposure was 19 ± 12.99 months. Results: After ten months of treatment a complete regression of chylothorax and chyloperitoneum occurred in 13 patients, and partial regression occurred in one patient. Treatment resulted in decrease of lymphangioma volume (1603.85 ± 2437.56 cm3 vs. 198.01 ± 315.43 cm3; p=0.00026), stabilisation of pulmonary function parameters such as (FVC, FEV1, TLC, DLCO), improvement of distance in 6MWT (514.46 ± 101.72 m vs. 526.79 ± 139.33 m; p=0.002), blood oxygenation (68.93 ± 13.6 mmHg vs. 78.23 ± 11.14 mmHg; p=0.04), and decrease of serum VEGF-D concentration (2591 ± 1085 pg/mL vs. 1274 ± 708 pg/mL; p=0.003). Conclusion: Sirolimus has proved to be an effective treatment of lymphangioma, chylous pleural and peritoneal effusions in LAM patients.
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