Tuberous sclerosis complex (TSC) is a rare, autosomal dominant disease, characterized by the presence of benign tumors in several organs, including the kidneys. Cystic disease and angiomyolipomas (AML) are the most frequent TSC-related renal lesions, which may be present since childhood. Renal AMLs, present in up to 80% of patients, may grow over time, leading to serious complications, such as bleeding and chronic kidney disease (CKD). mTOR inhibitors (mTOR-i) have been proved to be a safe and effective therapy for TSC patients, leading to AMLs regression, reducing their risk of bleeding and preserving renal function.
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Monich et al. (2024) studied this question.
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