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Published on: November 10, 2023
Prognostic role of pleural effusion or ascites in localized rhabdomyosarcoma
Daniela Di Carlo1, Andrea Ferrari2, Tiziana Toffolutti3
1Hematology/Oncology Division, Department of Women's and Children's Health, University of Padova, Padova, Italy.
Insights
Pleural effusion or ascites in children with rhabdomyosarcoma (RMS) may indicate a poor prognosis. Moderate to massive effusions significantly worsen outcomes, suggesting a need for targeted experimental studies in these high-risk pediatric cancer patients.
Area of Science:
- Pediatric Oncology
- Medical Imaging
- Clinical Research
Background:
- Pleural effusion and ascites are often considered poor prognostic indicators in pediatric rhabdomyosarcoma (RMS).
- Current treatment intensification for these cases lacks robust scientific validation.
- This study evaluates the prognostic significance of effusions in localized RMS.
Purpose of the Study:
- To investigate the prognostic role of pleural effusion or ascites at diagnosis in children with localized rhabdomyosarcoma (RMS).
- To determine if the presence and extent of effusion impact patient survival.
- To inform treatment decisions and clinical trial enrollment for pediatric RMS patients.
Main Methods:
- Retrospective review of radiological reports for 150 children with localized RMS enrolled in Italian Soft Tissue Sarcoma Committee protocols over 30 years.
- Assessment of effusion presence (pleural effusion or ascites) and extent (minimal, moderate, massive).
- Analysis of 5-year progression-free survival (PFS) rates based on effusion status and extent.
Main Results:
- Effusion was present in 21.3% of patients (14 pleural, 18 ascites).
- No significant difference in PFS for patients with any effusion versus no effusion (49.8% vs. 49.5%, P=.5).
- Moderate or massive effusions were associated with significantly lower PFS (36.8%) compared to minimal or no effusion (51.2%, P=.01).
- Patients with pleural effusion had a particularly poor 5-year PFS of 35.7%.
Conclusions:
- Moderate or massive effusions are unfavorable prognostic factors in pediatric rhabdomyosarcoma (RMS).
- The findings support the inclusion of these high-risk patients in experimental treatment studies.
- This research provides evidence-based rationale for intensified management in specific pediatric RMS subgroups.
Purpose:
The presence of pleural effusion or ascites at the time of diagnosis is generally considered a poor prognostic factor for children with rhabdomyosarcoma (RMS), and treatment is usually intensified despite the fact that there are no published studies to support this decision. We investigated the prognostic role of the presence of pleural effusion or ascites at diagnosis in patients with localized RMS consecutively enrolled in the Italian Soft Tissue Sarcoma Committee protocols over a 30-year period.
Methods:
We reviewed the radiological reports at diagnosis of 150 children with supradiaphragmatic and infradiaphragmatic RMS, noting any presence of effusion and its extent (minimal, moderate, or massive). All patients received intensive chemotherapy, surgery, and standard or hyperfractionated radiotherapy.
Results:
Effusion was identified in 32 children (21.3%), 14 with pleural effusion and 18 with ascites. As for its extent, 13 children presented with minimal, 12 with moderate, and 7 with massive effusion. The 5-year progression-free survival (PFS) rate was 49.8% (confidence interval [CI] 31.7-65.5) and 49.5% (CI 40-58.2) for patients with and without effusion, respectively (P = .5). When only patients with moderate or massive effusion were considered, however, their PFS was 36.8% (CI 16.5-57.5) versus 51.2% (CI 42.2-59.5) in patients with minimal or no effusion (P = .01). On the whole, patients with pleural effusion had a very poor outcome with a 5-year PFS of 35.7% (CI 13-59.4).
Conclusions:
The presence of moderate or massive effusion seems to be an unfavorable prognostic factor in children with RMS, and justifies their inclusion in experimental studies.
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