Maintenance Chemotherapy in Patients With High-Risk Rhabdomyosarcoma: Long-Term Survival Analysis of the European Paediatric Soft Tissue Sarcoma Study Group RMS
2005 Trial

Gianni Bisogno, Julia Chisholm, Raquel Hladun, Gian Luca De Salvo, Florent Guerin, Michela Casanova, Henry Mandeville, Rita Alaggio, Beatrice Coppadoro, Daniel Orbach, Andrea Ferrari, Rick van Rijn, Anne-Sophie Defachelles, Myriam Ben-Arush, Heidi Glosli, Maja Cesen, Johannes H.M. Merks and Veronique Minard-Colin. J Clin Oncol. 2025 Mar 7:JCO2402850. doi: 10.1200/JCO-24-02850. Online ahead of print.PMID: 40053891

The RMS2005 trial, conducted by the European Paediatric Soft Tissue Sarcoma Study Group (EpSSG), tested whether adding a long-term, low-dose chemotherapy treatment (maintenance chemotherapy) could help children with high-risk rhabdomyosarcoma (RMS), a rare and aggressive cancer. Patients were randomly assigned to either stop treatment after initial therapy (standard group) or continue with six additional months of maintenance chemotherapy (experimental group).

Earlier results showed that maintenance chemotherapy improved overall survival, but its effect on preventing cancer recurrence was unclear. Now, after more than 10 years of follow-up, researchers have confirmed that patients who received maintenance chemotherapy had better long-term survival. After 10 years, 83% of patients in the experimental group were still alive compared to 71% in the standard group. The findings confirm that maintenance chemotherapy with vinorelbine and low-dose cyclophosphamide provides a lasting survival benefit for high-risk RMS patients.