伊立替康联合替莫唑胺治疗儿童难治性、复发性髓母细胞瘤疗效观察

Clinical efficacy of irinotecan and temozolomide in treatment of refractory and recurrent child medulloblastoma

目的观察伊立替康联合替莫唑胺(IT)治疗儿童难治性、复发性髓母细胞瘤(MB)疗效。方法选取2015年7月～2017年12月在北京世纪坛医院住院的难治性、复发性MB患儿27例,均给予IT治疗:伊立替康总量300 mg/m2,静脉滴注,分2 d完成;替莫唑胺150 mg/(m2·d),口服,连用5 d;每4周为1个化疗周期;每2～3个周期接受全脑全脊髓MRI检查。治疗过程中监测患儿的血常规、肝肾功能、心肌酶等指标。随访至2018年10月,期间观察并记录患儿的无进展生存期、无进展生存率(PFS)、总体生存率(OS),进行疗效评价,疗效分为完全缓解(CR)、部分缓解(PR)、疾病稳定(SD)、疾病进展(PD),记录患儿的不良反应及其占比。结果截止到2018年10月,27例患儿中,CR 2例、PR 5例、SD 10例、PD 10例,化疗有效率(CR+PR)为25. 9%,疾病控制率(CR+PR+SD)为62. 9%。27例患儿的中位无进展生存期为6(1～12)个月,6个月、10个月PFS分别为74%、66. 7%,1年、2年OS分别为95%、87%。27例患儿中,腹泻发生率为29. 6%,3～4级骨髓抑制发生率为18. 5%,没有患儿因化疗不良反应死亡。结论 IT治疗儿童难治性、复发性MB疗效较好,可作为儿童难治性或复发性MB的治疗方案之一。

Objective To explore the clinical efficacy of irinotecan and temozolomide( IT) in the treatment of refractory and recurrent child medulloblastoma( MB). Methods Twenty-seven children with refractory and recurrent MB were included in Beijing Shijitan Hospital during the period of July 2015 to December 2017,all were given IT treatment: 300 mg/m2 irinotecan,intravenous drip,finished in 2 days,150 mg/( m^2·d) temozolomide,oral for five days,every 4 weeks as a chemotherapy cycle;receiving whole brain and spinal cord MRI inspection every 2-3 cycles. Blood routine examination,liver function,kidney function and myocardial enzyme of the patients were monitored in the course of treatment.Follow-up was conducted until Octboer 2018,and we observed and recorded the progression-free survival time,progressionfree survival( PFS),and overall survival( OS) to evaluate efficiency of treatment. Curative effect evaluation was divided into complete remission( CR),partial response( PR),stable disease( SD) and disease progression( PD),and the adverse reactions and their proportions were recorded. Results Until October 2018,there were 2 cases of CR,5 cases of PR,10 cases of SD,10 cases of PD,with the response rate( CR + PR) of 25. 9% and disease control rate( CR + PR +SD) of 62. 9%. In 27 patients,the median PFS was 6( 1-12) months,6-month and 10-month PFS was 74% and 66. 7%,and 1-year and 2-year OS was 95% and 87%,respectively. In 27 patients,the most frequent adverse reaction was diarrhea with the incidence rate of 29. 6%,followed by bone marrow suppression,with the incidence rate of 18. 5%,no child died from adverse reaction. Conclusion IT is effective in treatment of refractory or recurrent MB in children,and can be one of the treatment schemes for refractory and recurrent MB in children.