Background Previous studies have reported cyclophosphamide (CYC) to be active in the treatment of fibrosing alveolitis in its early, inflammatory stages. Akesson et al.¹ demonstrated, in CYC treated SSc patients with alveolitis, an effect on inflammation parameters and skin thickening.

Objectives To test, by a prospective 2-years trial, the efficacy and safety of low-dose oral CYC in the treatment of ?active? SSc whatever the disease manifestation underlying activity.

Methods SSc patients consecutively admitted were divided into 2 groups: group I with active disease (i.e. patients who had a new disease manifestation and/or a deterioration of a previously present one in the six months preceding the enrollment or who presented laboratory alterations indicating immune-inflammatory activity such as hypergammaglobulinemia and/or hypocomplementemia) and group II with inactive disease (i.e. patients with neither feature). Group I patients were treated with oral CYC (50 mg/daily) in addition to the previously administered symptomatic treatment; group II patients were only treated with symptomatic therapy. The following parameters (total skin score; ulcer healing; FVC; DLCO; EF; gammaglobulin levels; complement levels) and the severity scales recently proposed by Medsger et al.² were carried out at 12 and 24 months.

Results Out of the 45 recruited patients (42 F, 3M; aged 30 to 72 years, median age 49; 34 with lcSSc, 11 with dcSSc; disease duration ranging from 1 to 34, median 16 years) 22 were included in group I and 23 in group II. The study was discontinued for lack of efficacy in 5 out of the 19 group I patients and 8 out of the 19 group II patients who had at least the 12-months assessment (p > 0.05). No significant difference was detected between the 2 groups in any clinical, laboratory and instrumental parameters assessed, including Medsger?s severity scales. C3 and C4 significantly increased in the group I patients after 12 and 24 months; ESR decreased in group I patients only after 12 months.

Conclusion Our results suggest that CYC might be able to contrast the evolution of disease in a significant percentage of SSc active patients. The role as a disease-modifying drug of SSc as a whole must be further investigated.

References

1. Akesson, et al. Arthritis Rheum. 1994;37:729–35

2. Medsger TA, et al. J Rheumatol. 1999;26:2159–67