{"title":"婴儿恶性实体瘤的预后:单中心经验。","authors":"Burcu Tufan Taş, Nurşah Eker","doi":"10.3390/children12091276","DOIUrl":null,"url":null,"abstract":"<p><p><b>Background:</b> Malignant solid tumors diagnosed during the first year of life represent a rare but clinically significant subgroup of pediatric cancers. Their biological behavior, treatment responses, and prognosis differ substantially from tumors diagnosed in older children due to developmental immaturity and age-related therapeutic limitations. <b>Methods:</b> We retrospectively analyzed 88 infants diagnosed with malignant solid tumors before 12 months of age at a single tertiary center between March 2011 and March 2023. Demographic, clinical, pathological, and treatment data were collected. Overall survival (OS) was estimated by Kaplan-Meier analysis, and prognostic factors were evaluated using univariate and multivariate Cox regression models. <b>Results:</b> Of the 98 initially screened patients, 88 were eligible for analysis. The median age at diagnosis was 7 months, with a median follow-up of 42 months. The most common tumor locations were intra-abdominal (64.7%), brain (20.5%), and bone/soft tissue (12.5%). Neuroblastoma was the leading diagnosis (30.7%), with spontaneous regression observed in 29.6% of cases. Atypical teratoid rhabdoid tumor (ATRT) was the most frequent brain tumor (9.1%). The 5-year OS for the entire cohort was 78.3%. Brain tumors were associated with significantly higher mortality (HR 4.32, <i>p</i> = 0.01), while intra-abdominal tumors predicted improved survival (HR 0.31, <i>p</i> = 0.02). <b>Conclusions:</b> Infantile malignant solid tumors display heterogeneous clinical behavior and outcomes. While favorable results can be achieved in neuroblastoma and soft tissue sarcomas, brain tumors, particularly ATRT, remain a therapeutic challenge. Age-specific, risk-adapted treatment strategies and earlier detection are critical to improving survival and reducing long-term sequelae in this vulnerable population.</p>","PeriodicalId":48588,"journal":{"name":"Children-Basel","volume":"12 9","pages":""},"PeriodicalIF":2.1000,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12468199/pdf/","citationCount":"0","resultStr":"{\"title\":\"Outcome of Infantile Malignant Solid Tumors: A Single-Center Experience.\",\"authors\":\"Burcu Tufan Taş, Nurşah Eker\",\"doi\":\"10.3390/children12091276\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p><b>Background:</b> Malignant solid tumors diagnosed during the first year of life represent a rare but clinically significant subgroup of pediatric cancers. Their biological behavior, treatment responses, and prognosis differ substantially from tumors diagnosed in older children due to developmental immaturity and age-related therapeutic limitations. <b>Methods:</b> We retrospectively analyzed 88 infants diagnosed with malignant solid tumors before 12 months of age at a single tertiary center between March 2011 and March 2023. Demographic, clinical, pathological, and treatment data were collected. Overall survival (OS) was estimated by Kaplan-Meier analysis, and prognostic factors were evaluated using univariate and multivariate Cox regression models. <b>Results:</b> Of the 98 initially screened patients, 88 were eligible for analysis. The median age at diagnosis was 7 months, with a median follow-up of 42 months. The most common tumor locations were intra-abdominal (64.7%), brain (20.5%), and bone/soft tissue (12.5%). Neuroblastoma was the leading diagnosis (30.7%), with spontaneous regression observed in 29.6% of cases. Atypical teratoid rhabdoid tumor (ATRT) was the most frequent brain tumor (9.1%). The 5-year OS for the entire cohort was 78.3%. Brain tumors were associated with significantly higher mortality (HR 4.32, <i>p</i> = 0.01), while intra-abdominal tumors predicted improved survival (HR 0.31, <i>p</i> = 0.02). <b>Conclusions:</b> Infantile malignant solid tumors display heterogeneous clinical behavior and outcomes. While favorable results can be achieved in neuroblastoma and soft tissue sarcomas, brain tumors, particularly ATRT, remain a therapeutic challenge. Age-specific, risk-adapted treatment strategies and earlier detection are critical to improving survival and reducing long-term sequelae in this vulnerable population.</p>\",\"PeriodicalId\":48588,\"journal\":{\"name\":\"Children-Basel\",\"volume\":\"12 9\",\"pages\":\"\"},\"PeriodicalIF\":2.1000,\"publicationDate\":\"2025-09-22\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12468199/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Children-Basel\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.3390/children12091276\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"PEDIATRICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Children-Basel","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3390/children12091276","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 0
摘要
背景:在生命的第一年诊断出的恶性实体瘤是一种罕见但临床意义重大的儿科癌症亚组。由于发育不成熟和年龄相关的治疗限制,它们的生物学行为、治疗反应和预后与大龄儿童诊断的肿瘤有很大不同。方法:我们回顾性分析了2011年3月至2023年3月在单一三级中心诊断为12个月前恶性实体瘤的88名婴儿。收集了人口学、临床、病理和治疗数据。采用Kaplan-Meier分析估计总生存期(OS),采用单因素和多因素Cox回归模型评估预后因素。结果:在最初筛选的98例患者中,88例符合分析条件。诊断时的中位年龄为7个月,中位随访时间为42个月。最常见的肿瘤部位为腹腔内(64.7%)、脑(20.5%)和骨/软组织(12.5%)。神经母细胞瘤是主要的诊断(30.7%),在29.6%的病例中观察到自发消退。非典型畸胎体样横纹肌样肿瘤(ATRT)是最常见的脑肿瘤(9.1%)。整个队列的5年OS为78.3%。脑肿瘤与较高的死亡率相关(HR 4.32, p = 0.01),而腹内肿瘤预测生存率提高(HR 0.31, p = 0.02)。结论:婴儿恶性实体瘤表现出不同的临床行为和预后。虽然在神经母细胞瘤和软组织肉瘤中可以取得良好的结果,但脑肿瘤,特别是ATRT,仍然是一个治疗挑战。针对特定年龄、适应风险的治疗策略和早期发现对于提高这一弱势人群的生存率和减少长期后遗症至关重要。
Outcome of Infantile Malignant Solid Tumors: A Single-Center Experience.
Background: Malignant solid tumors diagnosed during the first year of life represent a rare but clinically significant subgroup of pediatric cancers. Their biological behavior, treatment responses, and prognosis differ substantially from tumors diagnosed in older children due to developmental immaturity and age-related therapeutic limitations. Methods: We retrospectively analyzed 88 infants diagnosed with malignant solid tumors before 12 months of age at a single tertiary center between March 2011 and March 2023. Demographic, clinical, pathological, and treatment data were collected. Overall survival (OS) was estimated by Kaplan-Meier analysis, and prognostic factors were evaluated using univariate and multivariate Cox regression models. Results: Of the 98 initially screened patients, 88 were eligible for analysis. The median age at diagnosis was 7 months, with a median follow-up of 42 months. The most common tumor locations were intra-abdominal (64.7%), brain (20.5%), and bone/soft tissue (12.5%). Neuroblastoma was the leading diagnosis (30.7%), with spontaneous regression observed in 29.6% of cases. Atypical teratoid rhabdoid tumor (ATRT) was the most frequent brain tumor (9.1%). The 5-year OS for the entire cohort was 78.3%. Brain tumors were associated with significantly higher mortality (HR 4.32, p = 0.01), while intra-abdominal tumors predicted improved survival (HR 0.31, p = 0.02). Conclusions: Infantile malignant solid tumors display heterogeneous clinical behavior and outcomes. While favorable results can be achieved in neuroblastoma and soft tissue sarcomas, brain tumors, particularly ATRT, remain a therapeutic challenge. Age-specific, risk-adapted treatment strategies and earlier detection are critical to improving survival and reducing long-term sequelae in this vulnerable population.
期刊介绍:
Children is an international, open access journal dedicated to a streamlined, yet scientifically rigorous, dissemination of peer-reviewed science related to childhood health and disease in developed and developing countries.
The publication focuses on sharing clinical, epidemiological and translational science relevant to children’s health. Moreover, the primary goals of the publication are to highlight under‑represented pediatric disciplines, to emphasize interdisciplinary research and to disseminate advances in knowledge in global child health. In addition to original research, the journal publishes expert editorials and commentaries, clinical case reports, and insightful communications reflecting the latest developments in pediatric medicine. By publishing meritorious articles as soon as the editorial review process is completed, rather than at predefined intervals, Children also permits rapid open access sharing of new information, allowing us to reach the broadest audience in the most expedient fashion.