Pilot study of anti-thymocyte globulin plus mycophenolate mofetil in recent-onset diffuse scleroderma.

To assess the safety and efficacy of anti-thymocyte globulin (ATG) followed by mycophenolate mofetil (MMF) in the treatment of diffuse scleroderma. A pilot study of 13 patients with recent-onset diffuse scleroderma was carried out. Patients received ATG for 5 days, followed by MMF for 12 months. We recorded adverse events, scleroderma skin score, hand contractures, EuroQol score, scleroderma functional assessment, pulmonary function studies, echocardiogram and plasma creatinine concentration. Mean skin score decreased during the study from 28 at baseline to 17 after 12 months of MMF (P<0.01). Hand contractures worsened during the study. Mean measurements of systemic disease remained stable. One patient died after a scleroderma renal crisis. Five patients developed serum sickness after ATG treatment, but this was controlled by corticosteroid therapy. MMF therapy was well tolerated. ATG and MMF appear safe in scleroderma. The improvement in skin score and the apparent stability of systemic disease during the study period suggest that controlled studies of these agents are justified.