CT characteristics predict the response to everolimus or sirolimus of renal angiomyolipomas in patients with tuberous sclerosis complex
CT characteristics predict the response to everolimus or sirolimus of renal angiomyolipomas in patients with tuberous sclerosis complex
复制标题
CT 特征预测结节性硬化症患者肾血管平滑肌脂肪瘤对依维莫司或西罗莫司的反应
DOI：
10.1007/s11255-019-02093-6
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发表时间：
2019-04-01
影响因子：
2
通讯作者：
Cai, Yi
中科院分区：
文献类型：
--
作者：
Wang, Wenda;Guo, Hao;Cai, Yi
目的 探讨依维莫司和西罗莫司治疗结节性硬化症（TSC）相关血管平滑肌脂肪瘤（AML）容量反应的影响因素。方法对我中心2014年4月至2017年11月期间接受mTOR抑制剂治疗的30例TSC-AML（依维莫司18例，西罗莫司12例）进行回顾性分析。对流行病学数据、治疗反应和影响因素进行了回顾和分析。将年龄、性别、是否与SEGA和/或LAM相关、血浆雷帕霉素浓度、基线时AML体积以及基线时最大横截面AML平均CT值进行分析。 结果 依维莫司组纳入18例32个病灶患者，西罗莫司组12例15个病灶。除受累侧外，两组基线特征差异均无统计学意义（P = 0.008）。依维莫司组的 AML 平均体积在基线时为 1000 +/- 1276 cm(3)，在治疗后 6 个月时为 633 +/- 1121 cm(3) (P < 0.001)；在依维莫司组中，在基线时为 1984 +/- 2861 cm(3)，在治疗后 6 个月时为 1733 +/- 2533 cm(3) (P = 0.001)。分别是西罗莫司组。依维莫司和西罗莫司组的 AML 平均体积减少分别为 55.56% +/- 23.79% 和 30.5% +/- 22.8% (P = 0.001)。逐步多元线性回归分析显示，影响依维莫司和西罗莫司治疗 TSC 相关 AML 短期容量反应的因素是基线时的 AML 容量（分别为 P < 0.001 和 0.038）和基线时的平均 CT 值（分别为 P < 0.001 和 0.020）。高CT值组体积缩小≥50%的发生率明显高于低CT值组（90.5% vs. 18.2%，P < 0.001）。结论 依维莫司每日10 mg治疗TSC-AML患者可能比西罗莫司每日2 mg更有效。基线时 AML 体积和平均 CT 值是影响依维莫司或西罗莫司治疗 TSC-AML 短期体积反应的因素。
Objective To investigate factors influencing the volume response of everolimus and sirolimus in tuberous sclerosis complex (TSC) associated-angiomyolipomas (AML).Methods A retrospective analysis of 30 cases of TSC-AML treated by mTOR inhibitors (everolimus 18 cases, and sirolimus 12 cases) between April 2014 and November 2017 at our center was carried out. Epidemiological data, therapeutic response and influence factors were reviewed and analyzed. Age, sex, associated with SEGA and/or LAM or not, plasma rapamycin concentration, AML volume at baseline, and mean CT value of AML in the maximum cross-section at baseline were analyzed as potential influencing factors.Results Eighteen patients with 32 lesions in everolimus group and 12 patients with 15 lesions in sirolimus group were included. There was no statistically significant difference of baseline characteristics except for involved side (P = 0.008) between two groups. The mean volume of AML was 1000 +/- 1276 cm(3) at baseline and 633 +/- 1121 cm(3) at 6 months after treatment (P < 0.001) in everolimus group, and 1984 +/- 2861 cm(3) at baseline and 1733 +/- 2533 cm(3) at 6 months after treatment (P = 0.001) in sirolimus group, respectively. The mean volume reduction of the AML in everolimus and sirolimus groups were 55.56% +/- 23.79% and 30.5% +/- 22.8% (P = 0.001). Stepwise multiple linear regression analysis revealed that factors influencing the short-term volume response of everolimus and sirolimus for TSC-associated AML were AML volume at baseline (P < 0.001 and 0.038, respectively) and mean CT value at baseline (P < 0.001 and 0.020, respectively). The rates of >= 50% volume reduction in high CT value group was much higher than that in low CT value group (90.5% vs. 18.2%, P < 0.001).Conclusions Everolimus at 10 mg daily might be more effective than sirolimus at 2 mg daily in treatment of patients with TSC-AML. AML volume and mean CT value at baseline were factors influencing the short-term volume response of everolimus or sirolimus for TSC-AML.