1979 年至 2005 年间神经母细胞瘤患儿生存率的提高：意大利神经母细胞瘤登记报告。

Improved survival of children with neuroblastoma between 1979 and 2005: a report of the Italian Neuroblastoma Registry.

机构信息

Epidemiology and Biostatistics Section, Scientific Directorate, Department of Hematology-Oncology, and Laboratories of Diagnostic Pathology and Clinical Chemistry, Giannina Gaslini Children's Hospital, Genova, Italy.

出版信息

J Clin Oncol. 2010 May 10;28(14):2331-8. doi: 10.1200/JCO.2009.24.8351. Epub 2010 Mar 29.

DOI:10.1200/JCO.2009.24.8351

PMID:20351331

Abstract

PURPOSE

To describe treatment, clinical course, and survival of a cohort of Italian patients with neuroblastoma.

PATIENTS AND METHODS

The study includes data from 2,216 children (age 0 to 14 years) diagnosed between 1979 and 2005. Overall survival (OS) was analyzed by clinical and biologic features at presentation and periods of diagnosis: 1979 to 1984, 1985 to 1991, 1992 to 1998, and 1999 to 2005. The relative risk of second malignant neoplasm (SMN) was assessed by the standardized incidence ratio (SIR), with the Italian population selected as referent.

RESULTS

Yearly patient accrual increased over time from 58 to 102. Patients age 0 to 17 months represented 45.6% of the total population, and their incidence increased over time from 36.5% to 48.5%. The incidence of stage 1 patients increased over time from 5.8% to 23.2%. A total of 898 patients (40.5%) developed disease progression or relapse, 19 patients developed SMN, and two patients developed myelodysplasia. The cumulative risk of SMN at 20 years was 7.1%, for an SIR of 8.4 (95% CI, 5.1 to 13.2). A total of 858 patients (39%) died (779 of disease, 71 of toxicity, six of SMN, and two of tumor-unrelated surgical complications). Ten-year OS was 55.3% (95% CI, 53.0% to 57.6%) and increased over time from 34.9% to 65.0%; it was significantly better for females and patients age 0 to 17 months at diagnosis, with extra-abdominal primary, and stage 1 and 2 disease. OS improved significantly over time in stage 1 and 3 patients. In patients with stage 4 disease, the improvement occurred between the first and second time cohorts (6.7% v 23.5%), but not afterward.

CONCLUSION

The outcome of children with neuroblastoma has progressively improved. Long-term survivors bear a significant risk of SMN.

摘要

目的

描述意大利神经母细胞瘤患者队列的治疗、临床过程和生存情况。

方法

本研究纳入了 1979 年至 2005 年间诊断的 2216 名（0 至 14 岁）儿童的数据。通过临床表现和诊断时期的生物学特征分析总体生存率（OS）：1979 年至 1984 年、1985 年至 1991 年、1992 年至 1998 年和 1999 年至 2005 年。通过标准化发病率比（SIR）评估第二恶性肿瘤（SMN）的相对风险，以意大利人群作为参照。

结果

患者入组人数逐年增加，从 58 例增至 102 例。0 至 17 个月龄的患儿占总人群的 45.6%，其发病率也逐年增加，从 36.5%增加到 48.5%。1 期患儿的比例也逐年增加，从 5.8%增加到 23.2%。共有 898 例（40.5%）患儿出现疾病进展或复发，19 例发生 SMN，2 例发生骨髓增生异常。20 年 SMN 的累积风险为 7.1%，SIR 为 8.4（95%CI，5.1 至 13.2）。共有 858 例（39%）患儿死亡（779 例死于疾病，71 例死于毒性，6 例死于 SMN，2 例死于与肿瘤无关的手术并发症）。10 年 OS 为 55.3%（95%CI，53.0%至 57.6%），且随着时间的推移从 34.9%增加到 65.0%；女性和诊断时 0 至 17 个月龄、非腹腔原发、1 期和 2 期的患儿 OS 更好。1 期和 3 期患儿的 OS 随着时间的推移显著改善。对于 4 期疾病患儿，在第一和第二队列之间（6.7%比 23.5%），但之后并未出现改善。