LOW - DOSE CYCLOPHOSPHAMIDE PULSES IN SYSTEMIC SCLEROSIS - A PRELIMINARY EFFICACY STUDY

Background: Cyclophosphamide (CYC) is currently used in the treatment of active interstitial lung disease in patients with systemic sclerosis (SSc). Two recent reports suggest a role of this drug (2 mg/kg/die per os) as a disease modifying agent in early diffuse SSc.Objectives: To assess the efficacy of low-dose (500 mg) CYC pulses in SSc patients with early diffuse disease.Methods: Eight patients with SSc (all females; aged 23-51 years, median 38) with early diffuse disease (disease duration ranging from 11-27 months, median 16.5) (Le Roy et al. 1988), underwent a prospective trial with low-dose (500 mg) CYC pulses administered at days 1-8-15 and subsequently every 28 days for 6 months - 1 year. Modified Rodnan skin score (mRss) and HAQ-disability index (d.i.) were investigated as outcome measures.Results: mRss declined from 18.6 ± 5.92 to 14.37 ± 4.56 at 6 months (P< 0.005) and 11.6 ± 1.34 at 12 months (P< 005). No significant difference was detected in HAQ-d.i. (T-0 0.96 ± 0.92; T-6 months 0.78 ± 0.74; T-12 months 0.48 ± 0.56), the absence of any significance depending on differences among patients in the behaviour of HAQ-d.i. over time: it decreased in 4 patients; increased in 2 patients and remained stable in the other two.Conclusion: Low-dose CYC pulses have been recently shown to be quite safe in SSc patients in whom orally administered CYC treatment is affected by a high frequency of side effects. The present study suggests that such CYC regimen may have a role in the treatment of early diffuse SSc. Controlled studies on large series are warranted.