High-dose Cyclophosphamide Without Stem Cell Rescue in Autoimmune Diseases: A Systematic Review

Abstract

High-dose cyclophosphamide without stem cell rescue is a radical therapy for refractory autoimmune diseases. The objective was to review the results of high-dose cyclophosphamide without stem cell rescue in autoimmune diseases. PubMed, Scielo, and Embase databases were systematically searched for articles on high-dose cyclophosphamide without stem cell rescue treating autoimmune diseases between 1966 and September 2022. Twenty-nine studies were identified, including a total of 404 patients. The diseases most studied were systemic lupus erythematosus (n=113), multiple sclerosis (n=99), aplastic anemia (n=86), and myasthenia gravis (n=33). Most authors used the posology of 50mg/kg/day over four days of cyclophosphamide associated with Mesna, prophylactic antibiotics, G-CSF (granulocyte colony-stimulating factor), and support of red blood cells, and platelet transfusion. The most common side effects were febrile neutropenia, alopecia, and gastrointestinal complaints. Regarding outcomes, most of the studies demonstrated improvement of the underlying autoimmune disease, some long-lived, but relapses and failures were also identified. In conclusion, high-dose cyclophosphamide without stem cell rescue is an effective option for treating severe autoimmune diseases. This procedure is relatively safe when the appropriate supportive care measures are taken.

Cite this article as: de Carvalho JF, Skare TL. High-dose cyclophosphamide without stem cell rescue in autoimmune diseases: A systematic review. Eur J Rheumatol. 2024;11(2):53-63.