采用HIT-2000方案治疗的41例髓母细胞瘤患儿疗效分析

Treatment of children with medulloblastoma according to HIT - 2000 protocol： a retrospective integrated clini- cal analysis of 41 patients

目的对41例髓母细胞瘤患儿给予HIT－2000方案治疗，分析其5年无病生存率和放化疗的不良反应，探讨适合国内儿童髓母细胞瘤的治疗方法。 方法2010年1月至2013年1月住院治疗的髓母细胞瘤患儿共41例，年龄1.3～15.5岁。所有患儿均为手术切除肿瘤后到首都医科大学附属北京世纪坛医院儿科采用HIT－2000方案治疗。综合分析患儿的放化疗不良反应，采用Kaplan－Meier法计算生存率、无事件生存（EFS）率和总体生存（OS）率。 结果截至2016年1月，随访时间1.1～6.1年，中位随访时间4.4年，其中肿瘤完全消失26例（63.4%），进展7例（17.1%），复发4例（9.7%），稳定1例（2.4%），骨髓转移2例（4.8%），继发第二肿瘤1例（2.4%）。3年、5年OS率分别为（78.0±6.5）%、（68.0±8.9）%；EFS率分别为（56.1±7.8）%和（53.7±7.8）%。3岁以上患儿生存分析结果显示：5年OS率为（75.6±7.7）%，EFS率为（60.06±8.3）%，较OS率偏高。随访期内所有患儿均未出现放射性脑坏死及放射性脊髓炎。放疗期间12例出现不同程度的恶心、呕吐、腹痛等胃肠道反应，34例出现血常规下降。化疗最常见不良反应为胃肠道反应和血细胞减少，15例由于粒细胞缺乏合并感染，其中10例为肺部感染，5例为肠道感染，其中金黄色葡萄球菌感染1例，大肠杆菌感染3例，经积极抗感染及对症治疗均痊愈。 结论HIT－2000方案适合国内髓母细胞瘤患儿手术后序贯治疗，且疗效显著，患儿可耐受。

Objective To retrospectively analyze the resuhs of 41 children with medulloblastoma which treated according to the HIT - 2000 protocol, and explore the suitable treatment for China children. Methods From January 2010 to January 2013,41 children （ 1 year and 4 months - 15 years and a half） were treated with pediatric HIT - 2000 protocol at Department of Pediatrics, Beijing Shijitan Hospital, Capital Medical University. Integrated analysis of the toxicity of radiotherapy and chemotherapy, calculated event - free survival （EFS） rate and overall survival （OS） rate using Kaplan - Meier method. Results As of January 2016, after a mean follow - up of 4.4 years （ 1.1 - 6.1 years） , 26 cases（63.4% ） complete responses （ CR）, 7 cases （ 17. 1% ） progressive, and 4 cases （ 9.7% ） relapses, 1 case （2.4%） stable,2 cases （4.8%） bone marrow metastasis, 1 case （2.4%） with the second brain tumor were ob- served. OS rate at 3 years and 5 years were （78.0 ：i： 6.5 ）% and （68.0 _＋ 8.9 ）%, respectively. EFS rate at 3 years and 5 years were （56.1 ± 7.8） % and （ 53.7 ±7.8 ） % , respectively. OS and EFS at 5 years of 3 years older children were （ 75.6 ± 7.7 ） % and （ 60.06 ± 8.3 ） %, which much higher than those of all children, respectively. During the follow - up period, all patients were not involved radiation - reduced brain necrosis and myelitis. Twelve children suffered variant degrees of nausea, vomiting, abdominal pain and other gastrointestinal reactions, 34 cases involved neutropenia or throm- bocytopenia. The most common side - effects of chemotherapy were gastrointestinal toxicity and cytopenia. Unfortunate- ly,15 children involved infection due to neutropenia, 10 cases with pneumonia and 5 cases with intestinal infection. Blood bacteria culture results showed 1 case with Staphylococcus aureus and 3 cases with E. coli positive. Conclusions Pediatric HIT- 2000 protocols are suitable for China children with medulloblastoma, and China children can endure the side - effect of radiotherapy and chemotherapy.