Purpose: We determined the efficacy of a combination of corticosteroids and mycophenolate mofetil for retroperitoneal fibrosis.
Materials and methods: We performed a prospective observational study of the treatment of 7 patients with biopsy proven retroperitoneal fibrosis. Patients were treated with 40 mg prednisone daily with a gradual taper over 6 months. Mycophenolate mofetil was administered at a starting dose of 1,000 mg twice daily and continued for 6 months following resolution of systemic symptoms and extubation of affected ureters. Outcomes included normalization of laboratory evidence of inflammation, regression of fibrosis by computerized tomography and the ability to discontinue ureteral stents.
Results: Seven patients were treated with mycophenolate mofetil and prednisone. Five of the 7 patients had bilateral ureteral obstruction and 1 had unilateral obstruction requiring ureteral stents. Baseline and followup laboratory values were C-reactive protein 8.9 and 1.3 mg/dl (p=0.07), hemoglobin 10.7 and 12.7 gm/dl (p=0.001), erythrocyte sedimentation rate 76 and 14.5 mm per hour (p=0.015) and serum creatinine 3.32 and 1.08 mg/dl (p=0.07), respectively. Six of the 7 patients had a mean 32% regression of the retroperitoneal mass on computerized tomography. Ten of the 11 obstructed ureters were free of obstruction following ureteral stent removal. The mean time to stent removal was 10.5 months. One patient had a distal ureteral stricture and continued to require decompression. There were no treatment related side affects.
Conclusions: Mycophenolate mofetil combined with prednisone was safe and efficacious in this small cohort of patients with retroperitoneal fibrosis. Larger trials are needed to confirm these results.