From the Department of Orthopedic Surgery, William Beaumont Army Medical Center (Dr. Wells, Dr. Eckhoff, and Dr. Polfer); the Department of Orthopedic Surgery, Texas Tech University Health Sciences Center El Paso (Dr. Wells, Dr. Eckhoff, Dr. Davis, Dr. Rajani, and Dr. Polfer); and Biostatistics and Epidemiology Consulting Lab, Texas Tech University Health Sciences Center El Paso, El Paso, TX (Dr. Singh).
J Am Acad Orthop Surg Glob Res Rev. 2024 Oct 21;8(10). doi: 10.5435/JAAOSGlobal-D-24-00281. eCollection 2024 Oct 1.
Bone and joint tumors are the third most common cause of pediatric cancer-related deaths in the United States. Although there have been improvements in survival rates among pediatric cancer patients over the past few decades, bone and joint cancers remain the exception. Considering current clinical trials involving novel targeted therapies, the establishment of updated mortality rates and predictors of survival for this cancer would be prudent. This investigation sought to determine updated 5-year survival rates and predictors of survival among pediatric Ewing sarcoma (ES) of bone treated within the United States.
The National Cancer Database was retrospectively inquired for all pediatric ES cases within the most updated bone and joint public use file available in September 2022. The reported data were truncated to only include patients with reported 5-year vital (ie, survival) status. Cox proportional hazard regression was conducted on both the truncated data and the entire cohort to validate the findings. The patients were then separated into alive versus deceased cohorts, and univariate regression analysis was done followed by multivariable regression of notable variables of interest.
Overall, an aggregated 5-year survival rate of 74.5% was found in the included patient cohort. Patients with localized cancer had a comparatively improved 5-year survival rate of 84.70% as opposed to those with macrometastatic disease on presentation with a survival rate of 50.4%. Patient demographic-, tumor-, and treatment-specific variables all demonstrated an effect on survival. The multivariable predictors of worse mortality were found to include older age, larger tumor size (>8 cm), macrometastatic disease on presentation, and positive surgical margins.
This analysis serves to establish updated survival rates of pediatric ES treated within the United States to set standards for comparison among future studies. Continued multi-institutional and international collaboration is needed to optimize current treatment results and develop novel targeted therapies.
在美国，骨骼和关节肿瘤是儿童癌症相关死亡的第三大常见原因。尽管在过去几十年中，儿童癌症患者的生存率有所提高，但骨骼和关节癌症仍然是个例外。考虑到目前涉及新型靶向治疗的临床试验，为这种癌症建立最新的死亡率和生存率预测因素将是谨慎的。本研究旨在确定美国治疗的儿童骨骼尤文肉瘤（ES）的最新 5 年生存率和生存率预测因素。
回顾性查询 2022 年 9 月可获得的最新骨骼和关节公共使用文件中的所有美国儿童 ES 病例的国家癌症数据库。报告的数据被截断，仅包括报告有 5 年生存（即生存）状态的患者。对截断数据和整个队列进行 Cox 比例风险回归，以验证发现。然后将患者分为存活组和死亡组，进行单变量回归分析，然后对有意义的变量进行多变量回归。
总体而言，纳入患者队列的 5 年总生存率为 74.5%。局部癌症患者的 5 年生存率相对提高，为 84.70%，而初诊时存在巨转移疾病的患者生存率为 50.4%。患者的人口统计学、肿瘤和治疗特异性变量均对生存产生影响。多变量预测死亡率较差的因素包括年龄较大、肿瘤较大（>8cm）、初诊时存在巨转移疾病和阳性手术切缘。
本分析旨在确定美国治疗的儿童 ES 的最新生存率，为未来研究提供比较标准。需要继续进行多机构和国际合作，以优化当前的治疗结果并开发新的靶向治疗方法。
