Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China.

Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China.
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依维莫司与西罗莫司治疗结节性硬化症相关血管平滑肌脂肪瘤:一项中国多机构回顾性研究

DOI:
10.1186/s13023-021-01932-z
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发表时间:
2021-07-03
影响因子:
3.7
通讯作者:
Cai Y
Cai Y
中科院分区:
医学2区
文献类型:
--
作者:
Luo C;Zhang YS;Zhang MX;Chen MF;Li Y;Qi L;Li HZ;Zu XB;Cai Y

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PurposeTo evaluate efficacy and safety of everolimus and sirolimus in patients with tuberous sclerosis complex-associated angiomyolipomas(TSC-AML).Materials and methodsWe performed a multi-institutional retrospective study of TSC-AML patients with oral everolimus 10 mg or sirolimus 2 mg per day for at least 3 months.血管平滑肌脂肪瘤的体积估计采用正交测量MRI或CT。根据美国国家癌症研究所不良事件通用术语标准评估不良事件(AE)。所有数据均用SPSS19.0软件进行统计学分析。随着用药时间的延长,两种药物的治疗效果更加显著。6个月和12个月后,依维莫司组患者的TSC-AML体积缩小比西罗莫司组更明显。依维莫司治疗的患者中超过一半的患者减少≥ 50%,其中约80%的患者减少≥ 30%,高于西罗莫司治疗的患者。关于安全性,有两个groups.ConclusionsBoth依维莫司和西罗莫司的不良事件的发生率没有显着差异是优秀的治疗选择TSC-AML。然而,依维莫司比西罗莫司具有更好的治疗效果,特别是在减少TSC-AML体积方面。因此,推荐依维莫司作为TSC-AML的首选治疗。
PurposeTo evaluate the efficacy and safety of everolimus and sirolimus in patients with tuberous sclerosis complex-associated angiomyolipomas (TSC-AML).Materials and methodsWe performed a multi-institutional retrospective study of TSC-AML patients treated with oral everolimus 10 mg or sirolimus 2 mg per day for at least 3 months. Angiomyolipoma volume was estimated using orthogonal measurements by MRI or CT. Adverse events (AEs) were assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events. All analyses were performed using SPSS 19.0 software.ResultsResponse rates were high in both groups. With the prolonged medication durations, the therapeutic efficacy of both agents became more significant. The TSC-AML volume reduction after 6 and 12 months was more pronounced in patients with everolimus than those with sirolimus. More than half of the patients treated with everolimus had ≥ 50% reduction, and approximately 80% of them had ≥ 30% reduction, which was higher than that in patients treated with sirolimus. Regarding safety, there was no significant difference in the incidence of AEs between the two groups.ConclusionsBoth everolimus and sirolimus are excellent therapeutic options for TSC-AML. However, everolimus has a better therapeutic efficacy than sirolimus, particularly in reducing TSC-AML volume. Everolimus is therefore recommended as the first choice of therapy for TSC-AML.
评估依维莫司治疗中国结节性硬化症相关肾血管平滑肌脂肪瘤的疗效:一项为期两年的试验
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