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Ask the Experts - Options for Recurrent Wegener's Granulomatosis

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Ask the Experts - Options for Recurrent Wegener's Granulomatosis
A 40-year-old woman diagnosed with cytoplasmic-antineutrophil cytoplasmic antibody (c-ANCA)-positive vasculitis was treated with cyclophosphamide (Cytoxan) and developed a diffuse interstitial pulmonary process that resolved with drug discontinuation. Two years later, hematuria and a renal biopsy led to a diagnosis of Wegener's granulomatosis. The woman was treated with azathioprine (Imuran), but she developed significant hepatic toxicity. She has been receiving sulfamethoxazole/trimethoprim (Bactrim), low-dose prednisone, and, recently, mycophenolate mofetil (CellCept).

She now presents with new pulmonary nodules and an increasing c-ANCA. Pulmonary evaluation does not suggest fungal disease, although the nodules are too small (5-7 mm, peripheral) for bronchoscopic biopsy. Serologic and purified protein derivative tests are negative. If this is recurrent Wegener's granulomatosis, what are my treatment options?

It is unfortunate that you don't have a biopsy from the lung lesion associated with cyclophosphamide therapy. If infection rather than a drug reaction had been diagnosed, I would have restarted the cyclophosphamide. Without that information, restarting would be a hard option.

You have also tried mycophenolate mofetil. It appears you have not tried methotrexate. This might be helpful, but I am cautious in that recommendation because I am unaware of any data suggesting that methotrexate is useful for the kidney manifestations of Wegener's. This takes you to the realm of trying drugs that may modulate the immune system but lack any good trials demonstrating their effectiveness.  You might consider using cyclosporine (Neoral) up to 3 mg/kg, monitoring blood pressure and kidney function very carefully, and hoping it will work. 

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