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Metastatic neuroblastoma in infants: are survival rates excellent only within the stringent framework of clinical
A Di Cataldo1, A Agodi2, J Balaguer3
1Unit of Paediatric Haematology and Oncology, Department of Clinical and Experimental Medicine, Hospital Policlinico, University of Catania, Via Santa Sofia 78, 95123, Catania, Italy.
Introduction:
SIOPEN INES protocol yielded excellent 5-year survival rates for MYCN-non-amplified metastatic neuroblastoma. Patients deemed ineligible due to lack or delay of MYCN status or late registration were treated, but not included in the study. Our goal was to analyse survival at 10 years among the whole population.
Materials And Methods:
Italian and Spanish metastatic INES patients' data are reported. SPSS 20.0 was used for statistical analysis.
Results:
Among 98 infants, 27 had events and 19 died, while 79 were disease free. Five- and 10-year event-free survival (EFS) were 73 and 70 %, and overall survival (OS) was 81 and 74 %, respectively. MYCN status was significant for EFS, but not for OS in multivariate analysis.
Conclusions:
The survival rates of patients who complied with all the inclusion criteria for INES trials are higher compared to those that included also not registered patients. Five-year EFS and OS for INES 99.2 were 87.8 and 95.7 %, while our stage 4s population obtained 78 and 87 %. Concerning 99.3, 5-year EFS and OS were 86.7 and 95.6 %, while for stage 4 we registered 61 and 68 %. MYCN amplification had a strong impact on prognosis and therefore we consider it unacceptable that many patients were not studied for MYCN and probably inadequately treated. Ten-year survival rates were shown to decrease: EFS from 73 to 70 % and OS from 81 to 74 %, indicating a risk of late events, particularly in stage 4s. Population-based registries like European ENCCA WP 11-task 11 will possibly clarify these data.
Insights
Ten-year survival rates for metastatic neuroblastoma patients show a slight decrease, highlighting the importance of MYCN status for event-free survival. Late events pose a risk, especially in stage 4s neuroblastoma.
Area of Science:
- Pediatric Oncology
- Neuroblastoma Research
- Clinical Trial Analysis
Background:
- The SIOPEN INES protocol demonstrated excellent 5-year survival for MYCN-non-amplified metastatic neuroblastoma.
- Patients ineligible for the study due to MYCN status or registration delays were treated but excluded from initial analysis.
Purpose of the Study:
- To analyze 10-year survival rates in the entire population of metastatic neuroblastoma patients treated under the INES protocol.
- To evaluate the impact of MYCN status on long-term outcomes in this cohort.
Main Methods:
- Retrospective analysis of Italian and Spanish metastatic INES patients' data.
- Statistical analysis performed using SPSS 20.0.
Main Results:
- Among 98 infants, 5-year event-free survival (EFS) was 73% and overall survival (OS) was 81%.
- 10-year EFS decreased to 70% and OS to 74%, indicating late event risk.
- MYCN status significantly impacted EFS but not OS in multivariate analysis.
Conclusions:
- Patients adhering to INES trial criteria exhibited higher survival rates than those with registration issues.
- MYCN amplification strongly influences prognosis, underscoring the need for universal MYCN testing.
- A decrease in 10-year survival rates suggests a risk of late events, particularly in stage 4s neuroblastoma.
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