Early-onset therapy with sirolimus helps to improve prognosis of patients with lymphangioleiomyomatosis (LAM)

Abstract

Introduction: LAM is a rare cystic lung disease that affects almost exclusively young women. Therapy with the mTOR-inhibitor sirolimus has been established approximately in year 2006. Lung transplantation has long been performed in patients with progressive clinical course. In Germany, analysis of clinical and lungfunctional data of a substantial number of patients has not so far been performed. Methods: Retrospectively, data of 48 female patients were followed up regarding transplantation as therapeutic solution at pneumology department of MHH over 2,7 (1,0-8,2) years. Transplant-free survival was shown by Kaplan-Meier-curves, comparing outcomes using log-rank test. Patients that did receive treatment with sirolimus have been compared to those without therapy. Here, differentiation between patients diagnosed with LAM before and after year 2006 has been conducted. Results: Mean age at time of investigation was 51,5 (47-62) years, whereas the mean age at time of diagnosis was 38 (33-47) years. Overall, 18 patients (38%) have undergone lung transplantation. Patients in therapy with sirolimus showed a mean FEV1-decrease of 22ml/year. Patients that did not receive sirolimus showed a mean FEV1-decrease of 74ml/year. Ten years after diagnosis, patients with sirolimus showed a transplant-free survival of 97% and differ with p=0,07 from patients without sirolimus and diagnosis after 2006 (60%) and with p=0,12 from patients without sirolimus and diagnosis before 2006 (57%). Conclusion: In this small cohort of patients with LAM sirolimus seems to help to prevent clinical progression and therefore prolongs time to transplantation.