Germline Genetic Testing and Survival Outcomes Among Children With Rhabdomyosarcoma: A Report From the Children's Oncology Group
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View abstract on PubMed
Summary
This summary is machine-generated.Germline cancer-predisposition variants (CPVs) are linked to worse outcomes in children with rhabdomyosarcoma, particularly embryonal histology. CPV testing may improve prognosis and guide treatment in future clinical trials.
Area Of Science
- Pediatric Oncology
- Cancer Genetics
- Clinical Research
Background
- Germline cancer-predisposition variants (CPVs) can influence cancer development and patient outcomes.
- Understanding the impact of CPVs in pediatric rhabdomyosarcoma is crucial for refining treatment strategies.
Purpose Of The Study
- To investigate the association between CPVs and outcomes in children diagnosed with rhabdomyosarcoma.
- To determine if CPVs impact overall survival (OS) and event-free survival (EFS) in this patient population.
Main Methods
- A cohort study analyzed data from 580 children newly diagnosed with rhabdomyosarcoma across 171 sites.
- Patients were assessed for CPVs in 24 rhabdomyosarcoma-associated cancer-predisposition genes (CPGs) and an expanded set of 63 autosomal-dominant CPGs.
- Overall survival (OS) and event-free survival (EFS) were analyzed using Kaplan-Meier and Cox regression models, stratified by tumor histology and fusion status.
Main Results
- Patients with CPVs in rhabdomyosarcoma-associated CPGs showed a trend towards worse EFS (48.4% vs 57.8%) and OS (53.7% vs 65.3%) compared to those without CPVs.
- Adjusted analyses revealed significantly worse OS for patients with CPVs (adjusted hazard ratio [AHR], 2.49; P = .002).
- Patients with embryonal rhabdomyosarcoma and CPVs had significantly worse EFS (AHR, 2.25; P = .007) and OS (AHR, 2.83; P = .002). Importantly, CPVs were associated with poor outcomes in fusion-negative rhabdomyosarcoma, comparable to fusion-positive cases.
Conclusions
- Germline CPV testing may serve as a prognostic tool for children with rhabdomyosarcoma, especially those with embryonal histology.
- The findings suggest that CPVs impact outcomes independently of second malignant neoplasms.
- Incorporating germline CPV testing into risk-based clinical trials could enhance prognostic accuracy and treatment stratification for rhabdomyosarcoma.
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