Successful mycophenolate mofetil therapy in nine patients with idiopathic retroperitoneal fibrosis.

Objective: To assess the therapeutic benefit of mycophenolate mofetil (MMF) in retroperitoneal fibrosis (RF).

Methods: MMF 2 g/day and prednisone 1 mg/kg were initiated in nine patients with radiological (9/9) and histological verification (2/9) of idiopathic RF. Out of nine patients, seven needed bilateral ureteral stenting due to extensive hydronephrosis.

Results: All patients experienced regression of radiological extension. Out of seven patients, five were free of ureteral catheters after a mean of 5.6 months and two remained on stenting due to secondary stenosis. Within 6 months mean creatinine and CRP fell from 2.5 to 1.2 mg/dl and from 4.0 to 1.4 mg/dl, respectively. MMF was discontinued after a mean of 27 months. Prednisone was tapered to zero after a mean of 7 months. Side-effects were urinary tract infections in 7/9 patients and impaired glucose tolerance in 3/9. No recurrence occurred after withdrawal of glucocorticoids and MMF in 7/9 patients after a mean overall follow-up of 55 months (range 12-120).

Conclusions: Treatment with MMF and glucocorticoids was successful in inducing partial or complete and lasting remission in RF. The results suggest the use of MMF as additional immunosuppressive option.