儿童Ⅳ期肝母细胞瘤52例的临床特点及预后

Clinical characteristics and prognosis of 52 cases with Ⅳ stage hepatoblastoma in children

目的总结儿童肝母细胞瘤（HB）Ⅳ期病例的临床资料，分析其诊疗特点及预后。方法分析2006年9月至2014年6月，首都医科大学附属北京同仁医院收治根据儿童癌症组织实体瘤协作组分期标准分为Ⅳ期的HB患儿52例，男30例，女22例；中位年龄1．91岁（1个月-12岁7个月）。主要采用化疗结合手术治疗，化疗方案采用美国儿童肿瘤协作组的规范性化疗。采用19．0软件进行统计分析，分析治疗后Ⅳ期HB患儿的化疗效果、复发率、复发后临床缓解率以及预后因素。结果1．年龄特点：0～12个月发病16例（30．76％），13～36个月患儿24例（46．15％），〉3～6岁9例（17．31％），〉6—12岁7个月3例（5．78％）。2．转移部位：52例患儿中，多处远处组织器官转移40例（76．92％）；临近组织器官转移12例（23．08％）。3．随访至2015年1月31日，中位随访时间13个月（3～79个月），3年无事件生存率32．69％（17／52例），3年总生存率61．53％（32／52例），病死率38．46％（20／52例）。4．在治疗中、治疗后存在复发率59．61％（31／52例）。31例复发病例经治疗，生存时间为（45．23±4．72）个月，95％可信生存区间为35．98～54．48个月；2年无事件生存率为23．07％（12／52例）；生存分析提示3年、5年总生存率分别为32．69％（17／52例）、15．38％（8／52例）。结论Ⅳ期HB病例虽然复发风险高、疗效相对较差，但综合病情制定个体化的综合诊疗方案仍具有较高的临床缓解率。应坚定患儿治疗的信心，综合诊疗以延长生存率、提高临床治愈率。

Objective To study the diagnostic and treatment characteristics and the prognosis of Ⅳ stage hepatoblastoma（HB） by summing up the clinical data of the disease. Methods Fifty - two cases of Ⅳ stage HB were collected at Beijing Tongren Hospital Affiliated to Capital Medical University from September 2006 to June 2014 and the clinical stage was confirmed according to Children＇s Cancer Group（ Pediatric Oncology Group）. There were 30 male cases and 22 female cases. The median age was 1.91 years old （ 1 month - 12 years and 7 months old）. The main therapy for hepatoblastoma was surgery and chemotherapy. The regimens of chemotherapy were based on Children Ontology Group （COG） scheme. The clinical efficacy, and relapse rate and recurrence rate and prognosis factors were analyzed by SPSS 19.0 software. Results （ 1 ） Age characters ： in 52 cases, 0 - 12 months sick infants in 16 cases （30.76%）, 13 -36 months old sick infants in 24 cases （46.15%）, 〉3 -6 years old sick children in 9 cases （ 17.31% ） ,and 〉 6 - 12 years old sick children in 3 cases accounting for only 5.78%. （2） Metastasis site included distant organs metastasis in 40 cases （76.92%）, and adjacent tissues and organs metastasis in 12 cases （ 23.08% ）. （ 3 ） Follow - up studies were conducted till January 31 st ,2015, the medium follow- up time was 13 months （3 -79 months） , the 3 - year free event survival rate（EFS） was 32.69% （ 17/52 cases） ,the 3 - year overall survival rate （OS） was 61.53% （32/52 cases） ,and the mortality was 38.46% （20/52 cases）. （4）In 52 cases,31 cases had relapse during or after treatment, accounting for 59.61%. Among in relapse cases, the average survival time was （ 45.23 ±4.72 ） months, and 95% confidence interval was 35.98 - 54.48 months. 2 - year EFS was 23.07% （ 12/52 cases）. The 3 - year and 5 - year OS in relapse cases was 32.69% （ 17/52 cases） ,and 15.38% （8/52 cases）. Conclusions Although the relapse risk of IV stage HB cases is high and the prognosis is relatively poor, the clinical remission rate can be improved by using personalized and comprehensive treatment methods through evaluating individual conditions. The comprehensive treatment can enhance the treatment confidence ,prolong survival time and improve the clinical efficacy.