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婴儿恶性实体肿瘤的治疗结果:单中心经验

Outcome of Infantile Malignant Solid Tumors: A Single-Center Experience.

作者信息

Tufan Taş Burcu, Eker Nurşah

机构信息

Department of Pediatric Hematology and Oncology, Pendik Training and Research Hospital, Marmara University, Istanbul 34899, Turkey.

出版信息

Children (Basel). 2025 Sep 22;12(9):1276. doi: 10.3390/children12091276.

Abstract

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. 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. 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, = 0.01), while intra-abdominal tumors predicted improved survival (HR 0.31, = 0.02). 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.

摘要

出生后第一年诊断出的恶性实体瘤是小儿癌症中一个罕见但具有临床意义的亚组。由于发育不成熟和与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出的肿瘤有很大不同。我们回顾性分析了2011年3月至2023年3月期间在一家单一的三级中心诊断出的88例12个月龄前患有恶性实体瘤的婴儿。收集了人口统计学、临床、病理和治疗数据。通过Kaplan-Meier分析估计总生存期(OS),并使用单变量和多变量Cox回归模型评估预后因素。在最初筛查的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。最常见的肿瘤部位是腹腔内(64.7%)、脑(20.5%)和骨/软组织(12.5%)。神经母细胞瘤是主要诊断(30.7%),29.6%的病例观察到自发消退。非典型畸胎样横纹肌样瘤(ATRT)是最常见的脑肿瘤(9.1%)。整个队列的5年OS为78.3%。脑肿瘤与显著更高的死亡率相关(HR 4.32, = 0.01),而腹腔内肿瘤预示生存期改善(HR 0.31, = 0.02)。婴儿恶性实体瘤表现出异质性的临床行为和结局。虽然神经母细胞瘤和软组织肉瘤可以取得良好结果,但脑肿瘤,尤其是ATRT,仍然是一个治疗挑战。针对特定年龄、风险适应的治疗策略和早期检测对于改善这个脆弱人群的生存和减少长期后遗症至关重要。

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