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TSC AML Everolimus mTOR First-Line — ESENeph MCQ

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HardChronic Kidney DiseaseTSC AML Everolimus mTOR First-LineESENeph

A patient with tuberous-sclerosis complex has bilateral renal angiomyolipomas. The largest is 5.1 cm and enlarging, but there is no active haemorrhage and renal function is preserved. What is the preferred kidney-preserving treatment?

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Correct answer: D — Everolimus through the TSC service with toxicity and response monitoring

Explanation lettering: D = shown as A · C = shown as B · E = shown as C · A = shown as D · B = shown as E

A is correct. A growing TSC-associated angiomyolipoma above 3 cm that is at risk of complications but does not require immediate surgery is an established indication for mTORC1 inhibition. Everolimus can shrink tumour burden while preserving renal parenchyma. B mistakes a benign AML for cancer and sacrifices kidney tissue. C is important for acute bleeding or selected refractory lesions but is not the preferred planned systemic strategy for bilateral disease. D risks haemorrhage and duplicates tissue risk when imaging is characteristic. Treatment requires monitoring for stomatitis, infection, pneumonitis, metabolic toxicity, proteinuria and drug interactions.

Reference: NHS England: everolimus for TSC-associated angiomyolipomas: https://www.england.nhs.uk/publication/everolimus-votubia-for-treatment-of-angiomyolipomas-associated-with-tuberous-sclerosis/