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Cyclophosphamide + topotecan is a combination chemotherapy regimen used primarily for the treatment of relapsed or refractory sarcomas, especially Ewing sarcoma and rhabdomyosarcoma. Cyclophosphamide is an oxazaphosphorine alkylating agent that disrupts DNA function by cross-linking guanine bases in DNA strands, leading to cell death. Topotecan is a topoisomerase I inhibitor that binds to the DNA-topoisomerase I complex and prevents religation of single-strand breaks during DNA replication, resulting in double-stranded breaks and apoptosis. This combination has demonstrated activity in pediatric patients with relapsed/refractory sarcomas and has been investigated as salvage therapy in adults with recurrent disease[1][6][8]. The regimen is typically administered intravenously over 5 days every 21 days.
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