Double-blind, placebo-controlled study of oral calcitriol for the treatment of localized and systemic scleroderma

Background: Various treatments including corticosteroids, nonsteroidal anti -inflammatory drugs, D-penicillamine, interferon gamma, cyclosporine, and c ytostatic drugs have been used with limited success in both morphea and sys temic sclerosis (SSc). Objective: We investigated the effect of treatment with oral calcitriol in patients with localized or systemic scleroderma. Methods: A randomized, double-blind, placebo-controlled study of 9 months' duration with a 6-month follow-up was performed at the Department of Dermat ology. A total of 27 patients (7 patients with SSc and 20 with morphea) wer e selected on a minimal skin score of 3 for patients with morphea and 12 fo r those with SSc. Each patient received calcitriol (0.75 mug/day for 6 mont hs plus 1.25 mug/day for 3 months) or placebo for 9 months. Efficacy parame ters included skin score, measurement of serum markers of collagen synthesi s and degradation and, additional for the patients with SSc, oral aperture measurements, lung function studies, and esophagus motility. Results: The skin score in patients with morphea after 9 months' treatment showed no significant difference between the placebo and calcitriol groups (mean percentage reduction [SD] in skin score in the placebo group was -29. 3 [57.9]; in the calcitriol group it was -19.4 [46.6]). The small group of patients with SSc was inadequate to allow us to draw any conclusions regard ing efficacy. No significant change was found in the serum markers of colla gen metabolism. Conclusion: In this study calcitriol was not more effective than placebo in patients with morphea. Because of the small group of patients with SSc tre ated, no conclusions regarding efficacy in SSc can be drawn.