Effects of sirolimus in lymphangioleiomyomatosis patients on lung cysts and pulmonary function: long-term follow-up observational study

This study investigates the long-term effects of sirolimus on lung cysts and pulmonary function in patients with lymphangioleiomyomatosis (LAM) and evaluates the suitability of CT as an imaging biomarker for LAM progression. Seventy-three female patients diagnosed with definite LAM were included, with 39 (53.4%) receiving sirolimus. Quantitative and qualitative CT scoring for lung cysts were conducted at various time points and compared. Aview software (Coreline Soft) was used for quantitative analysis. Results showed significant increases in cyst scores from baseline to follow-up after starting sirolimus and from baseline to the last follow-up, but no significant differences between scores at the start of sirolimus and the last follow-up in treated patients. Sirolimus treatment led to a significant improvement in diffusing capacity for carbon monoxide (DLCO). Significant correlations were found between cyst scores and pulmonary function tests (PFTs). The study concludes that sirolimus benefits LAM patients and CT is a useful tool for monitoring lung cysts.