首都医科大学学报 ›› 2019, Vol. 40 ›› Issue (2): 169-173.doi: 10.3969/j.issn.1006-7795.2019.02.003

• 肿瘤的免疫治疗与代谢 • 上一篇    下一篇

儿童肝母细胞瘤的临床特征和治疗效果评价

王希思1, 杨维2, 黄程1, 赵文1, 赵倩1, 段超1, 苏雁1, 金眉1, 张大伟1, 秦红2, 王焕民2, 马晓莉1   

  1. 1. 国家儿童医学中心 首都医科大学附属北京儿童医院血液肿瘤中心 儿童血液病与肿瘤分子分型北京市重点实验室 儿科学国家重点学科 儿科重大疾病研究教育部重点实验室, 北京 100045;
    2. 国家儿童医学中心 首都医科大学附属北京儿童医院肿瘤外科, 北京 100045
  • 收稿日期:2018-01-17 出版日期:2019-03-21 发布日期:2019-04-15
  • 通讯作者: 马晓莉 E-mail:mxl1123@vip.sina.com
  • 基金资助:
    国家科技重大专项(2017ZX09304029004),首都卫生发展科研专项(2018-2-2095)。

Clinical features and outcome evaluation for children with hepatoblastoma

Wang Xisi1, Yang Wei2, Huang Cheng1, Zhao Wen1, Zhao Qian1, Duan Chao1, Su Yan1, Jin Mei1, Zhang Dawei1, Qin Hong2, Wang Huanmin2, Ma Xiaoli1   

  1. 1. National Center of Children's Health;Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University;Beijing Key Laboratory of Pediatric Hematology Oncology;National Key Discipline of Pediatrics, Ministry of Education;MOE Key Laboratory of Major Diseases in Children, Beijing 100045, China;
    2. National Center of Children's Health, Oncology Surgery Department, Beijing Children's Hospital, Capital Medical University, Beijing 100045, China
  • Received:2018-01-17 Online:2019-03-21 Published:2019-04-15
  • Supported by:
    This study was supported by National Science and Technology Key Projects(2017ZX09304029004),Capital Health Development Scientific Research Project(2018-2-2095).

摘要: 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:31.6~2 200 000 μg/L(中位数192 274.5 μg/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。

关键词: 肝母细胞瘤, 儿童, 治疗效果, 多学科治疗

Abstract: 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 November 30, 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 stage Ⅱ, 34 cases in stage Ⅲ, and 7 cases in stageⅣ. The value of AFP was in the range of 31.6-2 200 000 μg/L(median data was 192 274.5 μg/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.

Key words: hepatoblastoma, child, curative effect, multidisciplinary treatment

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