一项针对小婴儿神经母细胞瘤期待观察作为一线治疗的前瞻性研究：一项儿童肿瘤协作组研究。
A prospective study of expectant observation as primary therapy for neuroblastoma in young infants: a Children's Oncology Group study.
机构信息
Departments of Surgery and Pediatrics, Texas Children's Hospital and Baylor College of Medicine, Houston, TX 77030, USA.
出版信息
Ann Surg. 2012 Oct;256(4):573-80. doi: 10.1097/SLA.0b013e31826cbbbd.
OBJECTIVE
To demonstrate that expectant observation of young infants with small adrenal masses would result in excellent event-free and overall survival.
BACKGROUND
Neuroblastoma is the most common malignant tumor in infants, and in young infants, 90% of neuroblastomas are located in the adrenal gland. Although surgical resection is standard therapy, multiple observations suggest that expectant observation could be a safe alternative for infants younger than 6 months who have small adrenal masses.
METHODS
A prospective study of infants younger than 6 months with small adrenal masses and no evidence of spreading beyond the primary tumor was performed at participating Children's Oncology Group institutions. Parents could choose observation or immediate surgical resection. Serial abdominal sonograms and urinary vanillylmandelic acid and homovanillic acid measurements were performed during a 90-week interval. Infants experiencing a 50% increase in the volume of the mass, urine catecholamine values, or an increase in the homovanillic acid to vanillylmandelic acid ratio greater than 2, were referred for surgical resection.
RESULTS
Eighty-seven eligible patients were enrolled: 83 elected observation and 4 chose immediate surgery. Sixteen observational patients ultimately had surgery; 8 had International Neuroblastoma Staging System stage 1 neuroblastoma, 2 had higher staged neuroblastoma (2B and 4S), 2 had low-grade adrenocortical neoplasm, 2 had adrenal hemorrhage, and 2 had extralobar pulmonary sequestration. The 2 patients with adrenocortical tumors were resected because of a more than 50% increase in tumor volume. The 3-year event-free survival for a neuroblastoma event was 97.7 ± 2.2% within the entire cohort of patients (n = 87). The 3-year overall survival was 100%, with a median follow-up of 3.2 years. Eighty-one percent of patients on the observation arm were spared resection.
CONCLUSIONS
Expectant observation of infants younger than 6 months with small adrenal masses led to excellent event-free survival and overall survival while avoiding surgical intervention in a large majority of the patients.
目的
证明对患有小肾上腺肿块的婴儿进行期待性观察将带来极佳的无事件生存率和总体生存率。
背景
神经母细胞瘤是婴儿中最常见的恶性肿瘤，而在小婴儿中，90%的神经母细胞瘤位于肾上腺。虽然手术切除是标准治疗方法，但多项观察结果表明，对于小于 6 个月且肿瘤尚未扩散至原发灶以外的婴儿，期待性观察可能是一种安全的替代方法。
方法
在参与儿童肿瘤学组机构的研究中，对 6 个月以下且无肿瘤扩散证据的小肾上腺肿块婴儿进行了前瞻性研究。父母可以选择观察或立即手术切除。在 90 周的时间内，对婴儿进行腹部超声和尿液香草扁桃酸和高香草酸测量。如果肿块体积增加 50%、尿液儿茶酚胺值增加或高香草酸与香草扁桃酸比值增加超过 2，则推荐进行手术切除。
结果
共纳入 87 名符合条件的患者：83 名患者选择观察，4 名患者选择立即手术。16 名接受观察的患者最终进行了手术：8 名患有国际神经母细胞瘤分期系统 1 期神经母细胞瘤，2 名患有较高分期的神经母细胞瘤（2B 和 4S），2 名患有低度肾上腺皮质肿瘤，2 名患有肾上腺出血，2 名患有肺隔离症。2 名患有肾上腺皮质肿瘤的患者因肿瘤体积增加超过 50%而接受了手术切除。整个患者队列（n=87）的神经母细胞瘤事件无事件生存率为 97.7±2.2%。3 年总生存率为 100%，中位随访时间为 3.2 年。观察组 81%的患者免于手术切除。
结论
对小于 6 个月且患有小肾上腺肿块的婴儿进行期待性观察可带来极佳的无事件生存率和总体生存率，同时避免了大多数患者的手术干预。
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