Cyclophosphamide and Topotecan in Relapsed and Refractory Pediatric Extracranial Solid Tumors: A Retrospective Analysis.
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OBJECTIVE: To assess the effectiveness and safety of cyclophosphamide and topotecan in children with relapsed or refractory extracranial solid tumors. METHODS: This study included children with relapsed or refractory extracranial solid tumors treated with cyclophosphamide (250 mg/m2/day) and topotecan (0.75 mg/m2/day) for 5 days, 3-weekly, between January 2012 and February 2024. Event-free-survival (EFS) and overall survival (OS) were estimated using the Kaplan-Meier method. RESULTS: Eighteen patients with median (range) age 6 (2-13) years (72% boys) with diagnoses of neuroblastoma (61%), Ewing sarcoma (22%), and rhabdomyosarcoma (17%), were analysed. Median of 4-cycles of chemotherapy was given, mostly as second-line (78%). Among 15 evaluable patients, responses were complete-response (CR, n = 1), partial response (PR, n = 3), stable disease (SD, n = 2), and progressive disease (n = 9); disease control rate (CR + PR + SD) 40%. The median EFS and OS were 3.65 months and 9.72 months, respectively, with 1-year EFS and OS rates of 33% and 45%. There was no treatment-related mortality. CONCLUSION: Cyclophosphamide-topotecan shows efficacy and acceptable safety in pediatric relapsed/refractory solid cancers, highlighting the need for better strategies to improve outcomes.