Brief communication: tamoxifen therapy for nonmalignant retroperitoneal fibrosis.

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.