100例肝母细胞瘤基于新危险分层的治疗结果分析

Treatment outcome of 100 patients with hepatoblastoma based on a new risk stratification

目的提出适合中国肝母细胞瘤(HB)患者的危险分层方法,探讨高危HB患者的新治疗方法。方法2014年9月至2018年9月,中山大学肿瘤防治中心和中山大学附属第一医院收治的年龄≤18岁的100例初治HB患者,根据临床分期、甲胎蛋白水平、病理分型等因素,分为极低危、低危、中危和高危4个危险层级。极低危患者手术后不行化疗,随访观察;低危患者术后用C5V方案(顺铂+氟尿嘧啶+长春新碱)化疗共4个周期;中危患者术前和术后行C5VD方案(顺铂+氟尿嘧啶+长春新碱+阿霉素)化疗,共6~8个周期;高危患者术前和术后行C5VD方案与IIV方案(异环磷酰胺+伊立替康+长春新碱)交替化疗,共8个周期。结果100例患者分为极低危2例,低危10例,中危51例,高危37例。化疗前有可评价病灶者83例,化疗后部分缓解65例,疾病稳定10例,疾病进展8例,一线化疗客观有效率为78.3%。中位随访20个月,复发和进展30例,死亡27例。全组患者2年无进展生存率为69.2%,2年总生存率为72.0%。其中极低危、低危、中危、高危的2年无进展生存率分别为100%、88.9%、75.3%和43.2%,2年总生存率分别为100%、100%、81.0%和44.8%。结论新的HB危险分层方法简便可行。根据新危险分层方法分层后,对患者采取不同的治疗策略,极低危、低危和中危HB可获得较好的预后,高危HB患者的生存率仍有待提高,需要探讨新的治疗策略。

Objective To provide the risk stratification method of hepatoblastoma(HB)suitable for implementation in China and explore the new treatment method for high-risk HB patients.Methods A total of 100 cases of children and adolescents under 18 years old with newly diagnosed HB in Sun Yat-sen University Cancer Center and Sun Yat-sen University First Affiliated Hospital from September 2014 to September 2018 were included.According to the clinical stage,AFP level,pathological subtype and other factors,patients were stratified into four groups:extremely low-,low-,intermediate-and high-risk.The patients at very low risk were treated with surgery only and followed-up.The patients at very low risk were treated with C5V(Cisplatin+5-Fluroracil+Vincristine)regimen for 4 courses.The patients at intermediate risk were treated with C5VD(Cisplatin+5-Fluroracil+Vincristine+Doxorubicin)regimen before and after surgery for 6-8 courses.The patients at high risk were treated with C5VD and IIV(ifoshamide+irinotecan+vincristine)alternately before and after surgery for 8 courses.Results One hundred patients were stratified into extremely low-risk,low-risk,medium-risk and high-risk groups for 2,10,51 and 37 cases,respectively.Eighty three cases had evaluable lesions before chemotherapy.Among them,65 patients achieved partial remission,stable disease and progressive disease were observed in 10,and 8 cases,respectively,with a response rate of 78.3%.During a median follow-up of 20 months,30 patients experienced tumor relapse or progression,and 27 of them died.The 2-years progression-free survival(PFS)and overall survival(OS)rates were 69.2%and 72.0%,respectively.The 2-years PFS rates of patients with extremely low risk,low risk,medium risk and high risk were 100%,88.9%,75.3%and 43.2%,respectively.The 2-years OS rates were 100%,100%,81.0%and 44.8%,respectively.Conclusions The novel HB risk classification is simple and feasible.With active comprehensive treatment,patients at extremely low-,low-and medium-risk have excellent outcomes.The survival rate of high-risk HB patients remains to be improved,and new treatment strategies need to be explored.