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.
Akesson, et al. Arthritis Rheum. 1994;37:729–35
Medsger TA, et al. J Rheumatol. 1999;26:2159–67