From Local to International Approach: Prognostic Factors and Treatment Outcomes in Neuroblastoma-A 30-Year...

Background/Objectives: Over the past three decades, significant progress has been made in understanding the biology of neuroblastoma. The integration of prognostic factors has facilitated risk stratification and the development of targeted treatment approaches. This study aims to analyze the outcomes of pediatric patients with neuroblastoma treated at a single oncology center over a 30-year period. Methods: This retrospective study analyzed data from patients aged 0-18 years with neuroblastoma, treated at the Department of Pediatric Hematology and Oncology in Bydgoszcz, Poland, between 1993 and 2023. The study endpoints included the 5-year probability of overall survival (pOS), event-free survival (pEFS), and relapse-free survival (pRFS), analyzed separately for low/intermediate- and high-risk groups. Results: Seventy-five patients met the inclusion criteria. Thirty-two children were categorized as high-risk patients and forty-three as low/intermediate risk. During the study period, outcomes in the low/intermediate-risk group improved significantly (the 5-year pOS 85.7% vs. 100.0%, p = 0.019; the 5-year pRFS 85.7% vs. 100.0%, p = 0.662; the 5-year pEFS 83.3% vs. 100.0%, p = 0.038). In the high-risk group, outcomes improved but did not reach statistical significance (the 5-year pOS 0.0% vs. 41.1%, p = 0.342; the 5-year pRFS 0.0% vs. 32.5%, p = 0.180; and the 5-year pEFS 0.0% vs. 21.5%, p = 0.537). Sixteen patients experienced relapse, of whom only three survived; stem cell transplantation at relapse significantly improved survival (OS 0.0% vs. 50.0%, p = 0.001). In the multivariable analysis, stage at diagnosis was a prognostic factor for pOS (HR 6.0; 95%CI 0.7-49.6, p = 0.096), while pelvic localization was a risk factor for pRFS (HR 3.0; 95%CI 0.8-10.5; p = 0.084). Conclusions: This analysis highlights significant advancements in the diagnosis and treatment of neuroblastoma. Nevertheless, outcomes for high-risk patients and those who experience relapse remain poor, underscoring the need for further therapeutic improvements.