The Cyclophosphamide Story: From Killer Gas to Life-saving Drug for Cancers and Inflammatory Rheumatic and Musculoskeletal Diseases (I-RMDs)

Indian Journal of Rheumatology · Published 2025-08-06 · DOI 10.1177/09733698251355347

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Abstract

Cyclophosphamide (CYC), a derivative of mustard gas, has been a cornerstone in treating life-threatening complications of various inflammatory rheumatic and musculoskeletal diseases (I-RMDs) since the mid-twentieth century, despite its well-documented adverse effects. The transformation of CYC from a chemical warfare agent in World War I to a life-saving drug is remarkable and crucial for rheumatologists to understand. This article offers a historical overview of the events and the scientists who were instrumental in this transformation, along with its key role in I-RMDs. Over the ensuing decades, numerous drugs have been developed to address conditions for which CYC was once the sole treatment option. Nevertheless, even in the third decade of the twenty-first century, CYC continues to serve as a dependable option for managing certain complex and challenging complications of I-RMDs. Rheumatologists, however, face a significant challenge when considering the initiation of CYC therapy. They must carefully weigh its proven efficacy against its potential toxicities. Furthermore, practical considerations such as the route of administration, dosage schedule and treatment duration must be guided by the best available evidence. Unfortunately, such information is often dispersed across decades of publications, with recommendations that are sometimes inconsistent and perplexing. This review delves into two primary aspects of CYC: its historical evolution and the practical considerations for its use in contemporary rheumatology practice.

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Publication details

Year
2025

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