Cyclophosphamide as disease-modifying therapy for scleroderma: pros and cons

Cyclophosphamide is widely used to treat severe manifestations of numerous autoimmune rheumatic diseases including systemic sclerosis. Based on recent data, immunological and inflammatory activation remain the primary therapeutic targets in systemic sclerosis. Other effective therapies targeting the fibrotic mechanisms have not been developed despite the increased understanding of the key pathogenic pathways. The two pivotal studies of cyclophosphamide in scleroderma-associated interstitial lung disease have rejuvenated interest in this chemotherapeutic agent in this disease. Although the studies suggest that the benefit is modest, identification of appropriate patients is central to management to harness its maximal potential clinical efficacy. This article provides a timely evaluation of the benefits and drawbacks of cyclophosphamide in systemic sclerosis