Low-dose intravenous cyclophosphamide in systemic sclerosis: an open prospective efficacy study in patients with...

To investigate the efficacy of a treatment with low-dose intravenous cyclophosphamide (CYC) and low-dose prednisone in early diffuse cutaneous systemic sclerosis (dcSSc).

Patients with dcSSc and a disease duration <24 months consecutively admitted to a tertiary centre underwent a prospective 1-year study. They were treated with i.v. CYC 500 mg/pulses, 10 mg prednisone equivalent, and supportive therapy. Modified Rodnan skin score (mRss), Health Assessment Questionnaire-Disability Index (HAQ-DI), forced vital capacity (FVC), and diffusing lung capacity for CO (DLCO) were assessed as outcome measures. In addition, the nine Medsger severity scale scores were evaluated.

mRss and DLCO significantly improved at both 6 (p = 0.002 and 0.012, respectively) and 12 months (p = 0.002 and 0.003, respectively). HAQ-DI showed a nearly significant reduction at 12 months (p = 0.06). Medsger's severity scores also improved for general condition (p = 0.001), peripheral vascular (p = 0.05), skin (p = 0.02), joint/tendon (p = 0.001), muscle (p = 0.05), and lung (p = 0.02). No treatment interruption was needed.

This preliminary study suggests a role for low-dose i.v. CYC in the treatment of early dcSSc. Controlled studies are warranted.