儿童腹膜后神经母细胞瘤56例临床分析

Clinical analysis of 56 cases of pediatric retroperitoneal neuroblastoma

目的总结儿童腹膜后神经母细胞瘤(NB)的临床特征和预后,提高对儿童腹膜后NB的认识。方法回顾性分析首都医科大学附属北京儿童医院血液中心2008年1月至2011年1月收治的腹膜后NB患儿的临床表现、实验室检查、治疗和随访等资料,评价近期和远期疗效,评估2年无病生存率(EFS)。结果 56例确诊腹膜后NB患儿纳入分析,男34例,女22例。发病中位年龄41.5(7～147)个月。病程中位数为1.7个月(6d至60个月)。Ⅱ期1例,Ⅲ期2例,Ⅳ期52例,Ⅳs期1例。高危53例,中危2例,低危1例。①起病时发热24例(42.8%),肢体疼痛18例(32.1%),腹部包块14例(25.0%),腹痛12例(21.4%),下肢无力3例(5.4%)。②49/56例(87.5%)LDH>240U.L-1,47/51例(92.2%)NSE>16.3ng.mL-1,44/53例(83.0%)24h尿VMA>30μmol,14/30例(46.7%)SF>1500ng.mL-1。③肿瘤原发于肾上腺23例(41.1%),原发于腹膜后25例(44.6%),同时原发于肾上腺和腹膜后2例(3.6%),胸腹联合6例(10.7%)。肿瘤包绕腹部大血管29/49例(59.2%)。27/53例(50.9%)瘤灶直径>10cm,瘤灶包绕腹部大血管的发生率显著增高(P=0.002)。LDH在瘤灶包绕腹部大血管患儿中显著升高(P=0.021),在瘤灶直径>10cm患儿中亦显著升高(P=0.017)。④47例(83.9%)伴骨髓受累,46例(82.1%)有不同程度的骨骼转移,6例(10.7%)伴有腹腔其他脏器转移,3例(5.4%)伴有远处淋巴结受累。⑤40例行原发或转移瘤病理活检,其中神经节母细胞瘤10例(25.0%),NB30例(75.0%)。⑥1例Ⅳs随诊观察,13例失访,42例采用BCH-NB高危方案规律化疗,16例治疗中肿瘤进展,其余26例完全缓解,继续随访中,中位随访时间19.5(3～52)个月,2年EFS为57.4%。结论儿童腹膜后NB发病年龄较大,临床多表现为发热、肢体疼痛和腹部包块。LDH在巨大瘤灶及瘤灶包绕腹部大血管的患儿中明显升高。病理学检查以NB多见,2年EFS为57.4%。

Objective To assess the the biological and clinical features of neuroblastoma and to improve the recognition of this tumor. Methods Patients diagnosed as neuroblastoma admitted from Jan. 2007 to Jan. 2011 were retrospectively analyzed for the clinical features, detection results and therapy. Follow-up was conducted every 3 months to evaluate short-term and long-term therapeutic effect. Results Totally 56 eligible patients （22 females, 34 males） were included. The median age of disease onset was 41 （7 -147） months. The median course of the disease was 1.7 （0.2 -60） months. 1 patient was in INSS stage Ⅱ , 2 in stage Ⅲ, 52 in stage Ⅳ and 1 in stage IVs, 53 patients were in high-risk, 2 were in moderate-risk. （1）24 cases（42.8% ） were with fever, 18 （32.1%） with extremity pain, 14 （25.0%） with abdominal mass, 12 （21.4%） with abdominal pain, 3 （5.4%） with paraparesis. （2）In 49/56 cases（87.5% ）LDH was more than 240 U · L^-1, in 47/51 （92.2%）NSE was more than 16.3 ng · mL^-1 , in 44/53 cases （83.0%）VMAZ24 hours urine was more than 30 μmol, in 14/30 cases （46.7%）SF was more than 1 500 ng · mL^-1. （3）Primary tumor site was on adrenal in 23 patients （41.1%） ,retroperitoneal neuroblastoma in 25 cases（44.6% ）, adrenal gland and retroperitoneal neuroblastoma in 2 cases（ 3.6% ） ,retroperitoneal neuroblastoma and thorax in 6 cases（ 10.7% ）. LDH was more likely elevated in patients with surrounding abdominal major vessels and the patients with huge mass （ P = 0. 021 ; P =0.017）. （4）Bone marrow was involved in 47 eases（83.9% ）. Bone metastasis was found in 46 eases（82. 1% ）. 6 cases（ 10. 7% ）had abdominal dissemination. Distant lymph node was involved in 3 cases（5.4% ）. （5）40 patients were with pathological results, 10 cases（25.0% ） with ganglioneuroblastoma, 30 cases（75.0% ） with neuroblastoma. （6）1 case in stage IVs was followedup, 13 cases were missed follow-up, 42 cases were treated by NB-N6 protocol. In 16 cases the disease progressed, 26 cases remained followed up, the median follow-up duration was 19.5 （3 - 52） months, 2-year EFS was 57.4%. Conclusions The age of disease onset was elder in patients with retroperitoneal neuroblastoma than in those with posterior mediastinum. The main clinical presentations were fever, extremity pain and abdominal mass. LDH was more likely elevated in patients with surrounding abdominal major vessels and the patients with huge mass. The most common subtype pathologically was neuroblastoma. Two-year EFS was 57.4%.