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Congenital rhabdomyosarcoma: A report from the European paediatric Soft tissue sarcoma Study Group
Gianni Bisogno1, Veronique Minard-Colin2, Myriam Ben Arush3
1Hematology Oncology Division, Department of Women's and Children's Health, University of Padova, Padua, Italy.
Insights
Congenital rhabdomyosarcoma (RMS) in infants is challenging but often presents favorably. Age- and weight-adjusted chemotherapy without radiation shows promising survival rates for these young patients.
Area of Science:
- Pediatric Oncology
- Rare Cancers
- Soft Tissue Sarcomas
Background:
- Congenital rhabdomyosarcoma (RMS) poses treatment challenges in infants.
- This study focuses on tumors diagnosed within the first two months of life.
Purpose of the Study:
- To analyze treatment protocols and outcomes for congenital RMS.
- To evaluate the efficacy of current therapeutic strategies in this specific population.
Main Methods:
- Retrospective analysis of 24 patients with congenital RMS enrolled in European paediatric Soft tissue sarcoma Study Group protocols.
- Treatment included chemotherapy with age- and weight-adjusted doses; radiotherapy was avoided.
- Favorable histology and localized disease were common, with complete resection in 10 patients.
Main Results:
- The 5-year event-free survival (EFS) was 75.0% and overall survival (OS) was 87.3%.
- Progressive disease was the primary reason for treatment failure.
- Most patients had favorable histology and localized disease, with minimal toxicity from chemotherapy.
Conclusions:
- Congenital RMS often presents as a favorable disease in newborns.
- Weight- and age-adjusted chemotherapy and avoidance of radiotherapy are effective, without compromising outcomes.
- This approach offers a viable treatment strategy for congenital RMS, minimizing long-term side effects.
Procedure:
Congenital rhabdomyosarcoma (RMS) represents a challenging disease due to its characteristics and the difficulties in delivering treatment in this immature population.
Methods:
We analyzed treatment and outcome of patients with congenital RMS, defined as tumor diagnosed in the first 2 months of life, enrolled in the European paediatric Soft tissue sarcoma Study Group protocols.
Results:
Twenty-four patients with congenital RMS were registered. All, except one patient (PAX3-FOXO1-positive metastatic RMS), had favorable histology and localized disease. Three patients had VGLL2-CITED2/NCOA2 fusion. Complete tumor resection was achieved in 10 patients. No radiotherapy was given. Chemotherapy doses were adjusted to age and weight. Only two patients required further dose reduction for toxicity. The 5-year event-free survival (EFS) and overall survival (OS) were 75.0% (95% confidence interval [CI] 52.6-87.9) and 87.3% (95% CI 65.6-95.7), respectively. Progressive disease was the main cause of treatment failure.
Conclusion:
Patients with congenital RMS presented with a favorable disease, allowing weight- and age-adjusted doses of chemotherapy and avoidance of irradiation, without compromising the outcome.
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