Long-term efficacy and safety of sirolimus therapy in patients with lymphangioleiomyomatosis

Abstract excerpt

Sirolimus has been confirmed to be effective for lymphangioleiomyomatosis (LAM), a rare multisystem neoplastic disease in women. The long-term effects of sirolimus treatment for LAM, however, are largely unknown. We aimed to analyze the long-term efficacy and safety of sirolimus therapy for LAM with 4-year follow-up. In total, 142 sporadic LAM patients who took sirolimus for 1–4 years were retrospectively...

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