儿童1、2期神经母细胞瘤临床研究

Long-term follow-up of stage 1-2 neuroblastoma

目的：分析1、2期神经母细胞瘤（NB）的临床特征、诊疗方案及远期疗效，为进一步改进治疗方案提供循证依据。方法回顾性分析1998年6月至2010年12月初诊并接受治疗的神经母细胞瘤1、2期患儿的临床资料。结果 NB患儿237例，1期24例，2期25例，1、2期患儿占所有NB患儿的20.7%（49/237）。诊断时的中位年龄25个月（2周~9岁），男29例，女20例。31例（63.6%）患儿因体检或影像学检查意外发现肿块。原发部位以后纵隔和腹部多见，分别为21例（42.9%）、22例（44.9%）。病理类型为预后良好者40例（81.6％）。尿香草苦杏仁酸（VMA）正常者32例（91.4％）。进行检测的患儿血LDH升高均不到正常值5倍。N-MYC扩增1例。10例患儿进入低危组治疗，仅行手术治疗。39例患儿进入中危组治疗，进行了手术及化疗综合治疗。所有49例患儿治疗结束时均获得非常好的部分缓解（VGPR），中位随访时间为60个月（22~148个月），未满5年失访者9例，中位随访时间为3个月（1-32个月）。全组2年、3年、5年无事件生存率（EFS）及生存率（OS）均为100%。结论1、2期神经母细胞瘤预后良好，应进一步减少化疗强度和时间。

Objectives To evaluate the clinical features, treatment scheme and long-term outcomes of stage 1、2 childhood neuroblastoma （NB）. Methods The retrospective study included 49 newly diagnosed NB stage 1、2 patients from June 1998 to December 2010. Clinical data and long-term outcomes were analyzed. Results Twenty-four patients with stage 1 NB and twenty patients with stage 2 NB were found among all 237 patients with NB enrolled in this study. The median age at diagnosis was 25 months（ 2 week to 9 year old）,29 males and 20 females. Thirty-one patients （63.6%） without symptoms were discovered with tumor by physical or imaging examination. Thorax and abdomen were the most common sites of primary tumor （21 and 22 cases, accounting for 42.9% and 44.9% of all patients, respectively）. Forty （81.6%） NB patients had favorable pathology classification. One patient was of MYCN amplification status. Urine vanilla mandelic acid was normal in 32 （91.4%） patients, and serum lactate dehydrogenase was less than five times of the normal value in all patients. Ten NB patients were treated ac-cording to the low-risk protocol who received surgery alone.Thirty-nine patients were treated according to intermediate-risk protocol who received both surgery and chemotherapy. All the patients achieved very good partial remission （100%）.The medi-an follow-up period was 60 months（22 months to148months）. Nine patients were lost after a follow up of 3 months in medi-an. The 2-、3-、5-year event free survival and overall survial of all 49 patients was 100%. Conclusions The prognosis for neu-roblastoma of stage 1、2 in this study was with 100%survival, which provides opportunity for further reduction of dosage and/or duration of episodes in chemotherapy.