Combined prednisone and mycophenolate mofetil treatment for retroperitoneal fibrosis: a case series.

BACKGROUND: Small case series suggest that a combination of mycophenolate mofetil and prednisone may be an effective treatment for patients with retroperitoneal fibrosis.

OBJECTIVE: To describe the outcomes of adults with retroperitoneal fibrosis who received a combination of prednisone and mycophenolate mofetil.

DESIGN: Prospective case series of patients followed between 1 April 2005 and 1 July 2009.

SETTING: Single tertiary care facility.

PATIENTS: 28 patients with retroperitoneal fibrosis.

INTERVENTION: Prednisone, 40 mg/d, tapered over 6 months, and mycophenolate mofetil, 1000 mg twice daily, for a mean of 24.3 months.

MEASUREMENTS: Clinical course, laboratory assessment, and measurement of periaortic mass. Mean follow-up was 1012 days, and no patients were lost to follow-up.

RESULTS: Systemic symptoms resolved in all patients; 89% had a 25% or greater reduction in periaortic mass. Elevated erythrocyte sedimentation rate and serum creatinine level and decreased hemoglobin level normalized in all patients. Disease recurred in 2 of 28 patients.

LIMITATION: This was a small case series.

CONCLUSION: Combined prednisone and mycophenolate mofetil therapy is a potentially effective treatment for retroperitoneal fibrosis that warrants evaluation in randomized trials.

PRIMARY FUNDING SOURCE: None.