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

Abstract

Objective

To determine whether interferon-α (IFNα) 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 IFNα (13.5 × 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 IFNα 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 IFNα −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 IFNα −8.2 versus placebo +1.3; P = 0.01) or the diffusing capacity for carbon monoxide (mean change IFNα −9.3 versus placebo +4.7; P = 0.002). Skin biopsy showed no significant decrease in collagen synthesis in the IFNα group, and no significant differences in the levels of procollagen peptides were seen between the 2 groups.

Conclusion

This study suggests that IFNα is of no value in the treatment of scleroderma, and that it may in fact be deleterious.