Objective: To assess the clinical features and long-term outcomes of neuroblastoma (NB) in children less than 18 months of age, so as to provide evidence for further improvement of treatment. Method: Clinical data(sex, age, stage, risk group, treatment response, follow-up, etc.) of 155 NB patients under age of 18 months from June 2000 to December 2015 in Shanghai Children's Medical Center were analyzed retrospectively. The clinical features were summarized and the long-term follow-up results were evaluated. The overall survival (OS) and event-free survival (EFS) were analyzed by using Kaplan-Meier method. Factors including age, stage, risk group, bone marrow and bone metastasis, N-MYC status and dehydrogenase(LDH) level were analyzed by Log-Rank test. Result: Totally 155 eligible patients (96 males, 59 females) were included. The median age of disease onset was 7 months (11 days to 18 months). There were 31 cases of stage 1, 19 cases of stage 2, 45 cases of stage 3, 38 cases of stage 4 and 21 cases of stage 4S. The median follow-up time was 36 months (range 4 to 189 months), the 3-year and 5-year EFS rate were 89.6% and 85.2% respectively and the 3-year and 5-year OS rate were 96.2% and 94.1%, respectively. A total of 15 recurrent or progressed cases were observed. The median time to first recurrence was 11 months (range 3 to 39 months), 6 cases eventually died. Second malignancy occurred in one patient. The patients who had relapsed disease within 12 months from initial diagnosis have much lower 3-year OS rate than those in whom the disease recurred 12 months later (25.7% vs. 83.3%, P=0.020). Although the number of chemotherapy courses in median-high risk group reduced from 8.6 courses to 7.5 courses after the revision in 2008, the survival rate showed no significant difference between before and after (5-year EFS 74.4% vs. 84.3%, 5-year OS 89.0% vs. 92.9%, both P>0.05). In patients with stage 1 and stage 2, the 3-year EFS of 34 cases with surgery alone and 16 cases accepted chemotherapy were both 100%. Age at diagnosis, stage, risk group, MYCN status, LDH level, bone marrow involvement and bone infiltration had significant impacts on prognosis(all P<0.05). Conclusion: Satisfactory outcomes could be achieved in neuroblastoma in children aged within 18 months; the prognosis was better in children at age less than 12 months compared with 12-18 months. MYCN amplification, LDH more than 5 times upper limit of normal range, bone marrow and bone infiltration were associated with worse prognosis.Excellent survival rates could be achieved in children with stage 1 and 2 disease within 18 month's old accepted surgery alone, chemotherapy or radiotherapy could be avoided in these patients so as to reduce long-term adverse reactions.
目的： 分析≤18月龄的神经母细胞瘤(NB)患儿的临床特点及远期随访结果。 方法： 回顾性分析155例2000年6月至2015年12月于上海儿童医学中心明确诊断为NB且诊断年龄≤18月龄的患儿的病例资料(包括性别、年龄、分期、危险度分组、治疗反应和随访情况等)，总结临床特点并评估远期随访结果，预后分析采用Kaplan－Meier生存分析法。对发病年龄、分期、危险度分组、骨髓和骨转移、N－MYC状态和乳酸脱氢酶(LDH)水平等利用Log－Rank检验进行单因素分析。 结果： 155例患儿，男96例，女59例，中位年龄7月龄(11 d～18月龄)。1期31例，2期19例，3期45例，4期38例，4S期21例。中位随访时间36(4～189)个月，3年和5年无事件生存率(EFS)分别是89.6%和85.2%，3年和5年总生存率(OS)分别为96.2%和94.1%。15例出现进展或复发，复发中位时间11(3 ～39)个月，其中6例最终死亡。1例发生第二肿瘤。诊断后12个月内复发的患儿较12个月后复发的患儿预后差(3年OS 25.7%比83.3%，P＝0.020)。2008年修订化疗计划后中高危组接受的化疗总疗程数平均由8.6个减少至7.5个，但生存率较前差异无统计学意义(5年EFS 74.4%比84.3%，5年OS 89.0%比92.9%，P均>0.05)。1期和2期34例未放、化疗的患儿和16例接受化疗的患儿3年EFS均为100%。发病年龄、分期、危险度分组、骨髓和骨转移、N－MYC状态和LDH水平明显影响预后(P均<0.05)。 结论： ≤18月龄的NB患儿预后总体较好，<12月龄的患儿的生存率较12～18月龄的高。诊断时存在骨髓或骨转移、N－MYC扩增和LDH超过5倍正常值为预后不良因素。1期和2期患儿单纯手术治疗预后较好，可避免进一步化疗或放疗，以减少远期不良反应。.
Keywords: Follow-up studies; Infants; Neuroblastoma.