Interferon-α does not improve outcome at one year in patients with diffuse cutaneous scleroderma -: Results of a randomized, double-blind, placebo-controlled trial

Objective, To determine whether interferon-alpha (IFN alpha) reduces the severity of skin involvement in early (<3 years) diffuse scleroderma. Methods. In a randomized, placebo-controlled, double-blind trial, 35 patients with early scleroderma received subcutaneous injections of either IFN alpha (13.5 x 106 units per week in divided doses) or indistinguishable placebo. Outcomes assessed were the modified Rodnan skin score, as determined by a single observer at baseline, 6 months, and 12 months, as well as data on renal, cardiac, and lung function. Pre- and posttreatment skin biopsy samples were analyzed and blood was obtained for assessment of procollagen peptide levels. Results. There were 11 withdrawals from the IFN alpha group and 3 from the placebo group due to either toxicity, lack of efficacy, or death. In the intent-to-treat analysis, there was a greater improvement in the skin score in the placebo group between 0 and 12 months (mean change IFN alpha -4.7 versus placebo -7.5; P = 0.36). There was also a greater deterioration in lung function in patients receiving active therapy, as assessed by either the forced vital capacity (mean change IFN alpha -8.2 versus placebo +1.3; P = 0.01) or the diffusing capacity for carbon monoxide (mean change IFN alpha -9.3 versus placebo +4.7; P = 0.002). Skin biopsy showed no significant decrease in collagen synthesis in the IFN alpha group, and no significant differences in the levels of procollagen peptides were seen between the 2 groups. Conclusion. This study suggests that IFN alpha is of no value in the treatment of scleroderma, and that it may in fact be deleterious.