Department of Pediatrics, Texas Children's Hospital, Section of Hematology-Oncology Baylor College of Medicine, Houston, Texas.
Departments of Biostatistics.
Pediatrics. 2022 Nov 1;150(5). doi: 10.1542/peds.2022-056918.
We aimed to clinically characterize the health, neurocognitive, and physical function outcomes of curative treatment of Wilms tumor.
Survivors of Wilms tumor (n = 280) participating in the St. Jude Lifetime Cohort, a retrospective study with prospective follow-up of individuals treated for childhood cancer at St. Jude Children's Research Hospital, were clinically evaluated and compared to age and sex-matched controls (n = 625). Health conditions were graded per a modified version of the National Cancer Institute's Common Terminology Criteria for Adverse Events. Standardized neurocognitive testing was graded by using age-adjusted z-scores. Impaired physical function was defined by age- and sex-matched z-scores >1.5 SD below controls. Modified Poisson regression was used to compare the prevalence of conditions and multivariable logistic regression to examine treatment associations.
Median age at evaluation was similar between survivors and controls (30.5 years [9.0-58.0] and 31.0 [12.0-70.0]). Therapies included nephrectomy (100%), vincristine (99.3%), dactinomycin (97.9%), doxorubicin (66.8%), and abdominal (59.3%) and/or chest radiation (25.0%). By age 40 years, survivors averaged 12.7 (95% confidence interval [CI] 11.7-13.8) grade 1-4 and 7.5 (CI: 6.7-8.2) grade 2 to 4 health conditions, compared to 4.2 (CI: 3.9-4.6) and 2.3 (CI: 2.1-2.5), respectively, among controls. Grade 2 to 4 endocrine (53.9%), cardiovascular (26.4%), pulmonary (18.2%), neurologic (8.6%), neoplastic (7.9%), and kidney (7.2%) conditions were most prevalent. Survivors exhibited neurocognitive and physical performance impairments.
Wilms tumor survivors experience a threefold higher burden of chronic health conditions compared to controls and late neurocognitive and physical function deficits. Individualized clinical management, counseling, and surveillance may improve long-term health maintenance.
本研究旨在对接受保肢治疗的 Wilms 肿瘤患者的健康状况、神经认知功能和身体功能结局进行临床评估。
我们对参与圣裘德儿童研究医院(St. Jude Children's Research Hospital)开展的回顾性研究、并前瞻性随访的 Wilms 肿瘤幸存者(n=280)进行了临床评估,并与年龄和性别匹配的对照组(n=625)进行了比较。健康状况按照国立癌症研究所(National Cancer Institute)的常见不良事件术语标准(Common Terminology Criteria for Adverse Events)的改良版本进行分级。采用年龄调整后的 z 分数对标准化神经认知测试进行分级。身体功能受损定义为年龄和性别匹配的 z 分数低于对照组 1.5 个标准差以上。采用改良泊松回归比较两组的患病情况,采用多变量 logistic 回归分析治疗相关性。
幸存者和对照组的中位评估年龄相似(30.5 岁[9.0-58.0]与 31.0[12.0-70.0])。治疗方法包括肾切除术(100%)、长春新碱(99.3%)、放线菌素 D(97.9%)、多柔比星(66.8%)、腹部(59.3%)和/或胸部放疗(25.0%)。在 40 岁时,幸存者平均有 12.7(95%置信区间[CI]:11.7-13.8)个 1-4 级和 7.5(CI:6.7-8.2)个 2-4 级健康状况,而对照组分别为 4.2(CI:3.9-4.6)和 2.3(CI:2.1-2.5)。最常见的 2-4 级健康状况包括内分泌(53.9%)、心血管(26.4%)、肺部(18.2%)、神经(8.6%)、肿瘤(7.9%)和肾脏(7.2%)疾病。幸存者存在神经认知和身体功能障碍。
与对照组相比,Wilms 肿瘤幸存者的慢性健康状况负担高 3 倍,且存在晚期神经认知和身体功能缺陷。个体化的临床管理、咨询和监测可能会改善长期的健康维护。