Friedman Danielle Novetsky, Chastain Katherine, Chou Joanne F, Moskowitz Chaya S, Adsuar Roberto, Wexler Leonard H, Chou Alexander J, DeRosa Amelia, Candela Joanne, Magnan Heather, Pun Shawn, Kahan Tamara, Wolden Suzanne L, Meyers Paul A, Oeffinger Kevin C
Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York.
Department of Epidemiology-Biostatistics, Memorial Sloan Kettering Cancer Center, New York, New York.
Pediatr Blood Cancer. 2017 Nov;64(11). doi: 10.1002/pbc.26562. Epub 2017 Apr 18.
Children, adolescents, and young adults treated for Ewing sarcoma (ES) are at risk for disease-related and treatment-related complications. We aimed to describe early and late overall mortality, cause-specific mortality, and key adverse health outcomes in a large, single-institutional cohort of patients with ES.
Patients with ES diagnosed at age less than 40 years and treated at Memorial Sloan Kettering between 1974 and 2012 were included. Overall survival was estimated using Kaplan-Meier methods. Cox proportional hazards were used to examine the association of clinical and pathologic variables with overall survival. Cause-specific mortality was evaluated with the cumulative incidence function accounting for competing risks.
Three hundred patients with ES (60.3% male; median age at diagnosis: 16.8 years [range: 0.3-39]; 30.0% with metastatic disease at diagnosis) were followed for a median of 7.8 years (range: 0.2-37). Five-year overall survival was 65.2% (95% confidence interval [95% CI], 59.8-71.1%) for the entire cohort; 78.6% for those with localized disease; 40.1% for those with isolated pulmonary metastases; and 28.1% for those with extrapulmonary metastases. In multivariable analysis, older age at diagnosis, minority race/ethnicity, and metastatic disease at diagnosis were associated with inferior survival. Ten-year cumulative incidence of relapse/progression was 40.1%, with eight late relapses occurring at a median of 6.3 years after diagnosis (range: 5-14). Seventeen patients developed subsequent neoplasms (treatment-related myelodysplastic syndrome/acute myelogenous leukemia = 9; solid tumors = 6; nonmelanoma skin cancer [NMSC] = 4). Excluding NMSC and melanoma in situ, the cumulative incidence of subsequent malignant neoplasms at 25 years was 15% (95% CI, 4.8-25.1%).
Patients with ES are at high risk for relapse/progression and second cancers.
接受尤因肉瘤（ES）治疗的儿童、青少年和年轻成人存在与疾病及治疗相关并发症的风险。我们旨在描述一个大型单机构ES患者队列中的早期和晚期总死亡率、特定病因死亡率以及关键不良健康结局。
纳入1974年至2012年间在纪念斯隆凯特琳癌症中心诊断为ES且年龄小于40岁的患者。采用Kaplan-Meier方法估计总生存率。使用Cox比例风险模型检验临床和病理变量与总生存率的关联。采用累积发病率函数评估特定病因死亡率，并考虑竞争风险。
300例ES患者（男性占60.3%；诊断时中位年龄：16.8岁[范围：0.3 - 39岁]；30.0%在诊断时患有转移性疾病），中位随访时间为7.8年（范围：0.2 - 37年）。整个队列的5年总生存率为65.2%（95%置信区间[95%CI]，59.8 - 71.1%）；局限性疾病患者为78.6%；孤立性肺转移患者为40.1%；肺外转移患者为28.1%。多变量分析显示，诊断时年龄较大、少数族裔以及诊断时患有转移性疾病与较差的生存率相关。复发/进展的10年累积发病率为40.1%，8例晚期复发发生在诊断后中位6.3年（范围：5 - 14年）。17例患者发生了后续肿瘤（治疗相关骨髓增生异常综合征/急性髓系白血病 = 9例；实体瘤 = 6例；非黑色素瘤皮肤癌[NMSC] = 4例）。排除原位NMSC和黑色素瘤后，25年后续恶性肿瘤的累积发病率为15%（95%CI，4.8 - 25.1%）。
ES患者复发/进展和发生第二原发癌的风险很高。
