Department of Medical Oncology, Dr. B.R.A. IRCH, All India Institute of Medical Sciences, New Delhi, India.
Department of Medical Oncology, Jawaharlal Institute of Post Graduate Medical Education and Research, Puducherry, India.
Pediatr Blood Cancer. 2024 Nov;71(11):e31268. doi: 10.1002/pbc.31268. Epub 2024 Aug 13.
Children ≤5 years of age with Ewing's sarcoma (ES) possibly have a distinct disease biology, data on which are scarce. We evaluated clinical features, outcomes, and prognostic factors of ES among children with age ≤5 years.
Children with ES registered between 2003 and 2019 were included. Baseline clinical and treatment details were retrieved from medical records. Prognostic factors were identified using multivariable Cox regression. Clinical features and outcomes of children ≤5 years were compared with those greater than 5 years by chi-square and log-rank tests. Propensity score-matched (PSM) analysis was done to evaluate the impact of age on survival in the metastatic and localized subgroups.
Out of the 859 patients, 86 (10%) were ≤5 years of age (median age 4 years, 60 males [69.8%]). The most common location was the extremities (37.2%), followed by thorax (27.9%) and head and neck (H&N) (22.1%); baseline metastases were seen in 25 patients (29.8%). The median event-free-survival (EFS) and overall survival (OS) were 25.6 and 68.7 months, respectively. Metastatic disease predicted inferior OS (hazard ratio [HR] = 2.54, p = .018) and EFS (HR = 2.47, p = .007], symptom duration ≤3 months predicted an inferior OS (HR = 2.17, p = .048). Compared to age greater than 5 years, younger children had more H&N and less pelvic primaries (p < .001) and lesser baseline metastases (p = .037). PSM analysis did not reveal any significant impact of age on OS in the metastatic (HR = 1.59, p = .29) or localized cohort (HR = 1.77, p = .09).
Children with ES ≤5 years of age have a distinct favorable clinical presentation. However, age is not an independent prognostic factor for survival outcomes when adjusted for confounders.
患有尤因氏肉瘤（Ewing's sarcoma，ES）的 5 岁以下儿童可能具有独特的疾病生物学特征，但相关数据非常有限。本研究旨在评估 5 岁以下儿童的 ES 临床特征、结局和预后因素。
纳入 2003 年至 2019 年期间登记的 ES 患儿。从病历中提取基线临床和治疗详细信息。使用多变量 Cox 回归识别预后因素。采用卡方检验和对数秩检验比较 5 岁以下和 5 岁以上患儿的临床特征和结局。通过倾向评分匹配（propensity score matching，PSM）分析评估年龄对转移和局部亚组生存的影响。
在 859 名患者中，86 名（10%）年龄≤5 岁（中位年龄 4 岁，60 名男性[69.8%]）。最常见的部位是四肢（37.2%），其次是胸部（27.9%）和头颈部（H&N，22.1%）；25 名患者（29.8%）存在基线转移。中位无事件生存（event-free survival，EFS）和总生存（overall survival，OS）分别为 25.6 和 68.7 个月。转移疾病预示着较差的 OS（风险比[hazard ratio，HR]为 2.54，p=0.018）和 EFS（HR 为 2.47，p=0.007），症状持续时间≤3 个月预示着较差的 OS（HR 为 2.17，p=0.048）。与年龄>5 岁的患儿相比，年龄较小的患儿 H&N 部位和骨盆原发灶较少（p<0.001），基线转移较少（p=0.037）。PSM 分析未发现年龄对转移（HR=1.59，p=0.29）或局部亚组（HR=1.77，p=0.09）的 OS 有显著影响。
5 岁以下 ES 患儿具有独特的良好临床表现。然而，在校正混杂因素后，年龄并不是生存结局的独立预后因素。
