Iloprost as cyclic five-day infusions in the treatment of scleroderma - Anopen pilot study in 20 patients treated for one year

Objective. To evaluate the safety and efficacy of cyclic intravenous ilopro st therapy in diffuse or limited scleroderma. Methods. Twenty patients, 14 women and 6 men with a mean age of 47.8+/-8.2 years, were given iloprost in fusions for six hours a day during five consecutive days, at a rate of 0.5 to 2 ng/kg/min. The course was repeated every three months for one year. Ef ficacy was evaluated based on a scleroderma skin lesion score, an ischemic lesion score, a well-being self-assessment score, and lung function tests i ncluding measurement of the diffusing capacity of the lung for carbon monox ide. Safety was assessed based on adverse event collection. Results. The sc leroderma skin lesion and ischemic lesion scores decreased significantly ov er the one-year treatment period, from 37.1+/-16.5 to 10.2+/-6.9 (P<0.001) and from 31.8+/-19.1 to 2.2+/-2.0 (P<0.05), respectively. The well-being se lf-assessment score also showed a significant improvement, from 71.4+/-16.5 to 15.0+/-6.6 (P<0.001). The diffusing capacity for carbon monoxide was de creased in 11 patients at baseline and showed a slight, non significant inc rease in these patients after the treatment period. No serious or persisten t side effects were recorded. Conclusion. Cyclic intravenous iloprost thera py was associated with improvements in skin changes and in general health, as well as with a slight increase in the diffusing capacity of the lung for carbon monoxide. Our data suggest that iloprost may act on some of the pat hogenetic mechanisms of scleroderma in It way that improves the course of t he disease.