儿童肝母细胞瘤的临床特征和治疗效果评价

Clinical features and outcome evaluation for children with hepatoblastoma

目的总结首都医科大学附属北京儿童医院血液肿瘤中心近10年诊治的肝母细胞瘤(hepatoblastoma,HB)患儿临床特点和治疗效果,结合随访资料,为探讨HB的合理诊疗策略,减少不良反应,提高患儿的无事件生存率提供依据。方法分析2009年2月至2018年5月,在首都医科大学附属北京儿童医院新诊断的、规律诊治,并坚持长期随访的HB患儿。采用以手术治疗、化学药物治疗和放射治疗的多学科联合诊治方案。化学药物治疗方案采用A方案(顺铂+长春新碱+氟尿嘧啶)以及B方案(顺铂+阿霉素)的联合化学药物治疗方案。记录患儿年龄、性别、病程、病初血清甲胎蛋白(alpha fetal protein,AFP)、临床分期、病理类型、治疗方法和治疗效果,随访至2018年11月30日。结果共收治51例患儿,其中6例患儿因在笔者医院治疗时间短、回当地治疗未纳入临床总结中; 3例患儿治疗前因肿瘤破裂出血等合并症死亡。余42例接受系统治疗并随访。中位年龄19(2～102)个月。临床分期:Ⅱ期1例,Ⅲ期34例,Ⅳ期7例。病初血清AFP:31. 6～2 200 000μg/L(中位数192 274. 5μg/L)。39例患儿中位随访时间36(6～115)个月。5年无事件生存率为74. 72%。患儿化学药物治疗后全血细胞计数下降不明显,化学药物治疗期间无严重感染发生。化学药物治疗期间无明确心脏毒性指征。仅1例患儿出现短期听力受损。结论手术联合化学药物治疗是HB的主要治疗手段,现行方案中阿霉素和顺铂剂量患儿耐受性好,5年无事件生存率较高,临床需要密切随访这些患儿的远期预后。

Objective To summarize the clinical characteristics and treatment effect of hepatoblastoma(HB)in Beijing Children’s Hospital in the past ten years,and further to provide the basis for reasonable diagnosis and treatment strategy to reduce the side effects and improve the event-free survival.Methods The patients who received multidisciplinary medical therapy,including surgery,radiotherapy and chemotherapy,and followed up regularly were enrolled into the group.The chemotherapy includes regimen A(vincristine+cisplatin+fluorouracil)and regimen B(cisplatin+doxorubicin).We recorded the age,sex,course of disease,initial serum alpha fetal protein(AFP),clinical stage,pathological type,treatment and treatment effect of all the patients.The cut-off data of follow-up was November30,2018.Results In total 51 cases admitted in our hospital,42 cases were admitted into the research group(six cases were treated in other hospitals,three cases died because of the tumor rupture before treatment).According to the cases of medical recorder in the group,the median age was 19 months(2-102 months);1 case in stageⅡ,34 cases in stageⅢ,and 7 cases in stageⅣ.The value of AFP was in the range of 31.6-2 200 000μg/L(median data was 192 274.5μg/L).The median follow-up was 36 months(6-115 months)in 39 cases,and the 5-year event-free survival(EFS)was 74.72%.After the chemotherapy,the whole blood cells decreased insignificantly.Neither severe infection nor cardio toxicity and hearing impairment occurred during chemotherapy.Conclusion Comprehensive treatment and management of surgery combined with chemotherapy is the main treatment for children with HB.The dosage of doxorubicin and cisplatin in the current regimen is tolerable,with fewer side effects,and the 5-year EFS is better.Long-term follow-up of these children is needed in the future.