背景:复发性和难治性横纹肌肉瘤(RRMS)需要新的治疗方法。RRMS的II期临床试验通常利用放射学反应作为主要活动终点,一种在RRMS中存在几个限制的方法。在这个分析中,我们旨在估计RRMS的无事件生存期(EFS)终点,该终点可用作未来研究的基准.
方法:我们在1997年至2016年期间对参加13项单药II期儿童肿瘤学组和传统组试验的RRMS患者进行了回顾性研究。所有纳入的试验均使用放射学响应作为其主要活动终点。6个月的EFS从试验入组时间估计,置信区间为95%。临床特征,包括注册试验,性别,年龄,种族,组织学,先前化疗的数量,并评估其对6个月EFS的影响。
结果:我们在13项试验中确定了175名患者。6个月EFS为16.8%(11.6%-22.8%)。根据年龄,6个月的EFS没有差异,性别,种族,或组织学。对于少于或等于两个先前治疗线的患者,6个月EFS改善的趋势无显着。对于参加了达到主要放射学反应终点的试验的患者,以及与获得稳定疾病的患者相比,获得完全或部分反应的患者。
结论:参加单药II期试验的RRMS预后较差。在单一代理试验中,RRMS的6个月EFS可用作未来单一代理II期试验的RRMS特定基准。
BACKGROUND: Novel therapies are needed for relapsed and refractory rhabdomyosarcoma (RRMS). Phase II clinical trials in RRMS have typically utilized radiologic response as the primary activity endpoint, an approach that poses several limitations in RRMS. In this analysis, we aimed to estimate an event-free survival (EFS) endpoint for RRMS that could be used as a benchmark for future studies.
METHODS: We performed a retrospective study of patients with RRMS enrolling on 13 single-agent phase II Children\'s Oncology Group and legacy group trials from 1997 to 2016. All included trials used radiographic response as their primary activity endpoint. Six-month EFS was estimated from time of trial enrollment with 95% confidence intervals. Clinical characteristics, including trial of enrollment, sex, age, race, histology, number of prior chemotherapies, and radiographic response were evaluated for their impact on 6-month EFS.
RESULTS: We identified 175 patients across 13 trials. The 6-month EFS was 16.8% (11.6%-22.8%). No differences were seen in 6-month EFS based on age, sex, race, or histology. There were nonsignificant trends toward improved 6-month EFS for patients with less than or equal to two prior lines of therapy versus higher than two, for patients enrolled on trials that achieved their primary radiographic response endpoint versus trials that did not, and for patients who achieved complete or partial response compared to those achieving stable disease.
CONCLUSIONS: The prognosis of RRMS enrolled on single-agent phase II trials is poor. This pooled 6-month EFS of RRMS on single-agent trials may be used as a RRMS-specific benchmark for future single-agent phase II trials.