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Good news for young RMS survivors: maintenance chemotherapy doesn't meaningfully raise the risk of developing a second cancer. A large European trial following 1,633 pediatric rhabdomyosarcoma patients found no significant difference in secondary malignancy rates between those who received maintenance chemo and those who didn't. Notably, nearly 30% of kids who did develop a second cancer had an underlying genetic syndrome — a finding that could reshape future screening recommendations.
Pediatric rhabdomyosarcoma (RMS) survivors have reassuring news: a major European study confirms that maintenance chemotherapy — a regimen of low-dose cyclophosphamide and weekly vinorelbine — does not significantly increase the risk of developing a second malignant neoplasm (SMN). The findings, published in Cancer, come from the EpSSG RMS 2005 trial, which previously showed that maintenance therapy improved survival in high-risk localized RMS patients.
Across 1,633 patients with a median follow-up of 9.3 years, only 27 (1.7%) developed an SMN. The 8-year cumulative incidence was similar between those who received maintenance chemo (1.95%) and those who didn't (1.07%), with no statistically significant difference. The most common second cancers were osteosarcoma, low-grade glioma, and hematologic malignancies.
Key Takeaways:
Why it matters: These results support the continued use of maintenance chemotherapy in high-risk pediatric RMS without significantly elevating secondary cancer risk. The high rate of underlying genetic syndromes among SMN patients also signals a clear need for routine germline cancer predisposition testing in this population.