Efficacy and limits of Sirolimus therapy in patients with Lymphangioleiomyomatosis: a retrospective study

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Aim: Lymphangioleiomyomatosis (LAM) is a rare multysistemic disease characterized by cystic lung destruction, renal angiomyolipomas (AMLs), and lymphangioleiomyomas. Sirolimus stabilizes lung function in LAM, but its long-term effects remain unclear. This study evaluates the long-term effect of Sirolimus on respiratory function. Methods: We retrospectively analyzed clinical and functional data from 236 patients with LAM referred to our centre from 2001–2021. A total of 112 patients met inclusion criteria. Sirolimus response was assessed at 3 and 5 years. A decline in FEV1<75 ml/year was defined as a positive response to therapy (responder patients), whereas a decline in FEV1≥75 ml/year was defined as a negative response (non-responder patients). Results: FEV1 increased by 60 ml/year in the first year (even if not statistically significant) and stabilized until year 3. Afterward, FEV1 declined by 100 ml/year (p=0.013). The distance covered during the 6-minute walk test showed no significant change. In 37% of patients treated for at least 5 years (n=71), the response to treatment was lost (non-responders). No statistically significant differences were found between responders and non-responders patients regarding the presence of TSC-LAM and AMLs, diagnosis in menopause, baseline lung function, or VEGF-D levels. Conclusion: Sirolimus shows a time-dependent effect, with initial stabilization followed by decline in a subset of patients. Long-term monitoring and further studies on additional therapies are needed.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.