Maintenance therapy with everolimus for subependymal giant cell astrocytoma in patients with tuberous sclerosis (the EMINENTS study)
Maintenance therapy with everolimus for subependymal giant cell astrocytoma in patients with tuberous sclerosis (the EMINENTS study)
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DOI：
10.1002/pbc.26347
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发表时间：
2017-06-01
影响因子：
3.2
通讯作者：
Mlynarski, Wojciech
中科院分区：
文献类型：
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作者：
Trelinska, Joanna;Dachowska, Iwona;Mlynarski, Wojciech
目的：结节性硬化症（TCS）和室管膜下巨细胞星形细胞瘤（SEGA）患者的治疗选择之一是依维莫司治疗，每天一次，每天一次，达到谷浓度5-15 ng/ml（标准治疗）。本研究的目的是评估减少剂量依维莫司（每周三次，每日剂量，如标准治疗-维持治疗）在一组先前使用标准剂量治疗至少12个月的患者中的疗效和安全性。材料和方法：10例符合纳入标准的tsc相关SEGA患者（6男4女，中位年龄14.23岁）纳入单臂前瞻性试验。所有患者随访至少12个月（中位12个月，范围12-24个月）。由经验丰富的放射科医生和客观的基于计算机的方法评估0,90,180和360天的肿瘤体积并进行比较。将维持治疗期间注意到的不良事件（ae）与标准依维莫司治疗期间观察到的ae进行比较。结果：SEGA体积在随后的时间点（第0、90、120和360天）之间的差异无统计学意义。未见肿瘤再生的临床症状。与标准治疗相比，ae在维持期间的严重程度和频率明显降低。结论：小剂量依维莫司维持治疗是TSC和SEGA患者完成标准治疗后的有效治疗选择，并可降低不良反应发生率。
Objective: One of the therapeutic options for patients with tuberous sclerosis (TCS) and subependymal giant cell astrocytoma (SEGA) is everolimus treatment once daily, every day, to attain trough concentrations of 5-15 ng/ml (standard treatment). The aim of this study was to evaluate the efficacy and safety of a reduced dose of everolimus (three times a week with a daily dose as in standard treatment-maintenance therapy) in a group of patients who were previously treated with standard dose for at least 12 months.Materials and Methods: Ten patients (six males, four females; median age 14.23 years) with TSC-related SEGA who met inclusion criteria were included into a single-arm, prospective trial. All the patients were followed over at least 12 months (median 12 and range 12-24 months). Tumor volumes from day 0, 90, 180, and 360 were evaluated by an experienced radiologist and an objective computer-based method and compared. Adverse events (AEs) noted during maintenance therapy were compared to the AEs observed during standard everolimus therapy.Results: The differences in SEGA volume between subsequent time points (day 0, 90, 120, and 360) were not statistically significant. No clinical symptoms of tumor regrowth were observed. AEs were significantly less severe and less frequent during maintenance compared with standard therapy.Conclusions: Maintenance therapy with reduced-dose everolimus is an effective therapeutic option for patients with TSC and SEGA after the completion of standard therapy and may moderate the rates of adverse effects.