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General Medicine · Respiratory medicine: lymphangioleiomyomatosis
Sample itemA 32-year-old woman presents with her second spontaneous pneumothorax, progressive exertional breathlessness and a milky pleural effusion. CT shows numerous thin-walled cysts evenly distributed through both lungs, and a renal angiomyolipoma. Which drug slows the decline in lung function?
Original practice item, not a recalled exam question. Nothing here is recorded.
The answer and the reasoning
D. Sirolimus
Lymphangioleiomyomatosis involves smooth muscle proliferation driven by mTOR activation, linked to tuberous sclerosis complex mutations. Sirolimus, an mTOR inhibitor, stabilises lung function. Chylous effusions and renal angiomyolipomas are typical associations.
Option by option
A. Pirfenidone
Pirfenidone is an antifibrotic used in idiopathic pulmonary fibrosis.
B. Methotrexate
Methotrexate has no proven role in this disease.
C. Cyclophosphamide
Cyclophosphamide is not effective in this cystic lung disease.
D. Sirolimus (correct)
Correct. Sirolimus inhibits mTOR, the pathway that is overactive in this disease.
Source note: Original NextPrep sample item — pending expert review. This item is original practice material awaiting independent expert review, so confirm important facts in a standard textbook.
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