Stankiewicz Joanna, Pogorzała Monika, Księżniakiewicz Piotr, Styczyński Jan
Department of Pediatric Hematology and Oncology, Collegium Medicum, Nicolaus Copernicus University Torun, Jurasz University Hospital, Sklodowskiej-Curie 9, 85-094 Bydgoszcz, Poland.
Children (Basel). 2025 Apr 19;12(4):525. doi: 10.3390/children12040525.
: 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. : 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. : 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%, = 0.019; the 5-year pRFS 85.7% vs. 100.0%, = 0.662; the 5-year pEFS 83.3% vs. 100.0%, = 0.038). In the high-risk group, outcomes improved but did not reach statistical significance (the 5-year pOS 0.0% vs. 41.1%, = 0.342; the 5-year pRFS 0.0% vs. 32.5%, = 0.180; and the 5-year pEFS 0.0% vs. 21.5%, = 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%, = 0.001). In the multivariable analysis, stage at diagnosis was a prognostic factor for pOS (HR 6.0; 95%CI 0.7-49.6, = 0.096), while pelvic localization was a risk factor for pRFS (HR 3.0; 95%CI 0.8-10.5; = 0.084). : 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.
在过去三十年中，人们对神经母细胞瘤生物学的理解取得了重大进展。预后因素的整合促进了风险分层和靶向治疗方法的发展。本研究旨在分析在单一肿瘤中心接受治疗的小儿神经母细胞瘤患者30年间的治疗结果。
这项回顾性研究分析了1993年至2023年期间在波兰比得哥什儿科血液学和肿瘤学部门接受治疗的0至18岁神经母细胞瘤患者的数据。研究终点包括总生存（pOS）、无事件生存（pEFS）和无复发生存（pRFS）的5年概率，分别针对低/中危组和高危组进行分析。
75名患者符合纳入标准。32名儿童被归类为高危患者，43名被归类为低/中危患者。在研究期间，低/中危组的治疗结果有显著改善（5年pOS为85.7%对100.0%，P = 0.019；5年pRFS为85.7%对100.0%，P = 0.662；5年pEFS为83.3%对100.0%，P = 0.038）。在高危组中，治疗结果有所改善，但未达到统计学意义（5年pOS为0.0%对41.1%，P = 0.342；5年pRFS为0.0%对32.5%，P = 0.180；5年pEFS为0.0%对21.5%，P = 0.537）。16名患者出现复发，其中只有3名存活；复发时进行干细胞移植显著提高了生存率（OS为0.0%对50.0%，P = 0.001）。在多变量分析中，诊断时的分期是pOS的一个预后因素（HR 6.0；95%CI 0.7 - 49.6，P = 0.096），而盆腔定位是pRFS的一个危险因素（HR 3.0；95%CI 0.8 - 10.5；P = 0.084）。
该分析突出了神经母细胞瘤诊断和治疗方面的重大进展。然而，高危患者和复发患者的治疗结果仍然很差，这凸显了进一步改善治疗方法的必要性。
