Brief communication: tamoxifen therapy for nonmalignant retroperitoneal fibrosis

Abstract

Background: Anecdotal case reports suggest tamoxifen as a possible treatment for retroperitoneal fibrosis, but a systematic assessment of its effect is not available.

Objective: To describe the course and outcomes of patients with nonmalignant retroperitoneal fibrosis treated with tamoxifen.

Design: Prospective, consecutive series.

Setting: Single tertiary care referral center.

Patients: 19 patients with nonmalignant retroperitoneal fibrosis treated with tamoxifen from April 1998 through April 2005.

Intervention: Tamoxifen, 20 mg orally twice daily.

Measurements: Clinical improvement, laboratory variables, and follow-up computed tomography (CT) and gallium scan findings.

Results: Fifteen patients reported substantial resolution of symptoms after a median treatment duration of 2.5 weeks. Erythrocyte sedimentation rate and C-reactive protein also improved. Gallium scanning at follow-up showed incomplete disappearance of pathologic gallium-67 activity. Repeated CT scanning showed slow but steady mass regression in 14 of 15 clinical responders. Five patients failed treatment, including 1 patient who improved clinically. Disease recurred in 1 patient who responded to reintroduction of tamoxifen. One patient developed reversible hepatitis.

Limitations: This small observational study did not have a control group.

Conclusion: Tamoxifen may be a viable therapeutic option in the treatment of retroperitoneal fibrosis.