AIM: To assess the antitumor activity and safety profile of topotecan plus cyclophosphamide regimen in patients with relapsed/refractory paediatric type tumors and to analyze the progression free survival (PFS) and overall survival (OS) of these patients. MATERIALS AND METHODS: The details of patients of recurrent paediatric type tumors, treated over a period of consecutive five years from 2014 to 2018, with topotecan and cyclophosphamide regimen, were analyzed retrospectively. The patients should have had already received at least a previous chemotherapy regimen and had either a disease progression or relapse before being considered for the TC regimen. RESULTS: A total of 51 patients (median age 12yrs with 39 males and 12 females) received the TC regimen. The median number of cycles was 3 (range, 2–8). The response rate was: CR 0%, PR 35.3%, and disease stabilization (SD) 21.6 %. The median duration of PR in the patients was 7m (range4m -34m) and the median duration of disease stabilization was 6m (range 4m -16m). The median PFS and OS were 4m and 8m respectively. CONCLUSION: Topotecan plus cyclophosphamide is a well tolerated regimen for recurrent/ refractory paediatric type tumors, both in paediatric and adult populations, showing a good response rate. But, the duration of response (PFS) and OS is dismal.
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