儿童横纹肌肉瘤临床治疗评估

An evaluation of clinical treatment in children with rhabdomyosarcoma

目的分析评价横纹肌肉瘤(RMS)的临床治疗及预后。方法回顾分析2009年12月至2016年12月收治的31例RMS患儿的临床资料。结果 31例患儿中男19例、女12例,发病中位年龄42.2(7.2~109.9)个月;Ⅰ期2例,Ⅱ期6例,Ⅲ期16例,Ⅳ期7例。均采用上海儿童医学中心RS-2009方案化疗,随访至2017年5月1日。2例患儿治疗疗程中失访,1例停药复发后失访,中位随访时间39.3个月。Ⅰ期患儿均完全缓解并长期存活;Ⅱ期复发2例;Ⅲ期复发4例;Ⅳ期复发继而死亡3例,治疗无效进展死亡1例。Ⅰ、Ⅱ、Ⅲ期患儿3年无事件生存率(EFS)(61.8±12.9)%,3年总生存率(OS)100%;Ⅳ期3年EFS和OS均为(42.9±18.7)%。未放疗组3年EFS(37.7±14.2)%,正规放疗组(82.1±11.7)%。结论RS-2009方案对Ⅰ、Ⅱ和Ⅲ期儿童RMS疗效尚可,但对Ⅳ期尚需进一步优化;放疗可以显著提高EFS。

Objectives To investigate and evaluate clinical treatment and prognosis of children with rhabdomyosarcoma（RMS）. Methods A total of 31 cases of children（19 males and 12 females） diagnosed as RMS in Children＇s Hospital of Nanjing Medical University from December 2009 to December 2016 were followed up, with a median age of onset at 42.2 months（ 7. 2~109.9 months）. Prognosis of patients with RMS was clinically investigated. Result Among the 31 cases, there were 2 cases with stage Ⅰ, 6 cases with stage Ⅱ, 16 cases with stage III and 7 cases with stage IV. Protocol RS-2009 of Shanghai Children＇s Medical Center was used to treat these patients until May 1, 2017. Two cases were lost to follow up during the treatment course, one case lost after replase. Median of the follow-up time is 39. 3 months. After high intensive treatment, two cases with stage Ⅰ achieved complete remission and long-term survival, two cases with stage Ⅱ and four cases with stage Ⅲ recurred, three cases with stage Ⅳ recurred and died soon, one with stage Ⅳ did not response to treatment and progressed to death. The 3-year event free survival（EFS） of the cases in stage Ⅰ, Ⅱ and Ⅲ was（61.8%±12.9%）, the 3-year overall survival（OS） of them was 100%, while the 3-year EFS and OS both were（42.9±18.7%） for the cases with stage Ⅳ. The 3-year EFS of the cases with no radiotherapy was 37.7%±14.2%, while it was 82.1%±11.7% for those with regular radiotherapy. Conclusions The protocol RS-2009 of SCMC in the treatment of RMS in stage Ⅰ, Ⅱ and Ⅲ can get a curative effect, but it need to be improved and optimized to the cases with stage Ⅳ. Radiotherapy can highly increase EFS.