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Clinical Characteristics and Survival Outcomes in a Cohort of Pediatric Rhabdomyosarcoma Patients: The Impact of Risk-Adapted Therapy
by
Yanhua Li
†, 
Yangyang Jiao
†,
Xuelian Liao
,
Jingbo Shao
,
Ting Zhang
,
Can Huang
,
Jingwei Yang
* and
Shayi Jiang
* Department of Hematology and Oncology, Shanghai Children’s Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai 200040, China
*
Authors to whom correspondence should be addressed.
†
These authors contributed equally to this work.
Cancers 2026, 18(11), 1848; https://doi.org/10.3390/cancers18111848
Submission received: 17 April 2026
/
Revised: 30 May 2026
/
Accepted: 2 June 2026
/
Published: 4 June 2026
(This article belongs to the Special Issue Rare Tumors in Children: Trends in Diagnosis, Treatment and Prevention (2nd Edition))
Simple Summary
Rhabdomyosarcoma is a type of cancer that arises in the muscles and soft tissues, most commonly affecting children. While treatment has improved, outcomes can vary greatly, especially if the cancer has spread. This research was conducted to understand the clinical features, treatment effects, and key prognostic factors of pediatric rhabdomyosarcoma, and to compare two common treatment protocols. We aimed to identify factors that can help improve survival and guide clinical practice. Our study shows that tumor metastasis and complete surgical removal significantly affect patient survival, and delayed surgery is a safe and effective option for tumors in complex locations. These findings can help clinicians optimize risk stratification and multidisciplinary treatment, thereby improving the prognosis of children with this tumor.
Abstract
Background: Rhabdomyosarcoma (RMS) is the most common pediatric soft tissue sarcoma, with survival dependent on risk stratification and multimodal therapy. This single-center study explored the clinical characteristics, treatment outcomes and prognostic factors of pediatric RMS to optimize local management. Methods: Data of RMS patients treated at Shanghai Children’s Hospital from 2011 to 2024 were analyzed to estimate event-free survival (EFS) and overall survival (OS), and factors associated with survival. Patients received the Rs-99 (pre-2019) regimen or a modified Rs-2018 (post-2019) regimen. Results: A total of 76 RMS patients were identified. The 5-year EFS and OS for the entire cohort were 71.1% (95% CI, 62.3% to 83.3%) and 72.4% (95% CI, 64.0% to 84.5%), respectively. No statistically significant differences were observed in EFS and OS between the Rs-2018 and Rs-99 regimens. Univariate survival analysis indicated that metastasis was associated with prognosis: the 5-year EFS and OS of patients with metastatic disease were 35.0% (95% CI, 18.3% to 57.6%) and 40.0% (95% CI, 27.3% to 75.3%), while both the 5-year EFS and OS of patients with localized disease reached 83.9% (95% CI, 69.3% to 93.6%). Primary tumor resection status was a key prognostic factor for patients with localized disease, with a 5-year EFS of 100% for R0 resection and 46.7% (95% CI, 25.2% to 74.0%) for R2 resection. For patients with localized disease, EFS was comparable between those who underwent delayed primary excision (DPE) and upfront resection. Conclusions: Outcomes for patients with metastatic RMS remain poor. For those with localized disease, primary tumor resection status correlates with improved EFS and OS; additionally, DPE represents a feasible therapeutic option for localized RMS involving complex anatomical sites.
