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Long-term outcomes with non-dose dense chemotherapy for Ewing sarcoma – a follow up of the cohort treated with EFT-2001 protocol

7 Oct 2025
Badira Cheriyalinkal Parambil, Girish Chinnaswamy, Maya Prasad, Venkata Rama Mohan Gollamudi, Ajay Puri, Ashish Gulia, Sajid Qureshi, Prakash Nayak, Manish Pruthi, Siddhartha Laskar, Nehal Khanna, Jifmi Jose Manjali, Amit Janu, Sneha Shah, Nilendu Purandare, Mukta Ramadwar, Poonam Panjwani, Bharat Rekhi, Pallavi Rane, Shripad Banavali

Collaborative trials of co-operative groups have currently established interval compressed chemotherapy (ICC) as the standard of care, though there are concerns regarding the application of the same in low middle-income countries (LMICs). This study is a retrospective analysis of the long-term outcomes of a follow-up cohort (n = 200), constituted by patients (<15 years) with Ewing sarcoma (ES) treated with curative intent (including localised and metastatic patients) during January 2013–June 2017 on a non-dose dense chemotherapy protocol, EFT-2001. Local therapy was planned at 9–12 weeks of therapy and was delivered in all but three patients who had events before local control. At a median follow-up of 97 months (95%CI:91–103 months), 7-year event-free survival (EFS) and overall survival (OS) of the whole cohort were 55% (95%CI:49%–63%) and 69% (95%CI:63%–76%), respectively. Seven-year EFS and OS for the localised cohort were 60% (95%CI:53%–69%), 73% (95%CI:66%–80%) and for metastatic cohort were 37% (95%CI:24%–55%) and 53% (95%CI:39%–72%), (p = 0.003, p = 0.015), respectively. Non-relapse mortality was 8% (n = 16). Anthracycline dose, axial location, poor histological necrosis and older age group were associated with adverse outcomes. Cardiotoxicity was reported in 13%, with one-third developing symptomatic cardiac dysfunction. Long-term outcomes for children with ES treated on a non-dose dense chemotherapy protocol, in the setting of a higher treatment-related mortality, have relatively fair outcomes, though suboptimal compared to the ICC approach. ICC could be introduced in a phased manner in high-risk subsets in LMICs with better resources and an active nutritional rehabilitation and supportive care programme, while EFT-2001 protocol still could be a practical solution in resource-constrained settings.

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