Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex.
Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex.
复制标题
低剂量依维莫司维持治疗与结节性硬化症相关的肾血管平滑肌脂肪瘤
DOI：
10.3389/fmed.2021.744050
复制
发表时间：
2021
影响因子：
3.9
通讯作者：
Cai Y
中科院分区：
文献类型：
--
作者：
Luo C;Ye WR;Zu XB;Chen MF;Qi L;Li YL;Cai Y
目的：评估低剂量依维莫司维持治疗对既往接受标准剂量治疗至少6个月的结节性硬化症相关肾血管平滑肌脂肪瘤（TSC-RAML）患者的安全性和疗效。材料与方法：2018年4月至2019年4月，中南大学湘雅医院共入组了24例确诊TSC的患者。所有患者在标准剂量依维莫司诱导治疗后接受低剂量依维莫司维持治疗至少6个月。患者还接受了TSC 1/TSC 2基因检测，并在3、6、12、18和24个月进行随访。使用实体瘤疗效评价标准（RECIST，版本1.1）标准监测患者RAML缓解，同时根据美国国家癌症研究所不良事件通用术语标准（CTCAE，版本4.0）评估不良事件（AE）。P < 0.05是使用SPSS 19.0进行的所有分析的显著性水平。结果如下：所有24例患者均存在TSC 1/TSC 2基因突变，所有患者在最初6个月诱导治疗期间均实现了TSC-RAML体积的显著减少，并在低剂量维持治疗期间表现出体积稳定，无任何TSC-RAML再生长的情况。与标准治疗相比，维持治疗期间不良事件（AE）的严重程度和频率显著降低。结论：低剂量依维莫司维持治疗代表了在至少6个月的全剂量诱导治疗后实现TSC-RAML控制的有效方法，并且可能与依维莫司相关AE频率和严重程度的降低相关。
Objective: To assess the safety and efficacy of low-dose everolimus maintenance therapy for tuberous sclerosis complex-related renal angiomyolipoma (TSC-RAML) patients that had previously undergone standard-dose treatment for a minimum of 6 months. Materials and Methods: In total, 24 patients with a definitive TSC diagnosis were enrolled from April 2018 – April 2019 at Xiangya Hospital, Central South University. All patients underwent low-dose everolimus maintenance therapy following standard-dose everolimus induction therapy for a minimum of 6 months. Patients additionally underwent TSC1/TSC2 genetic testing, And they were followed-up at 3, 6, 12, 18, and 24 months. The Response Evaluation Criteria in Solid Tumors (RECIST, version 1.1) criteria were used to monitor patient RAML responses, while adverse events (AEs) were assessed as per the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE, version 4.0). P < 0.05 was the significance level for all analyses, which were performed using SPSS 19.0. Results: TSC1/TSC2 gene mutations were present in all 24 patients, all of whom achieved a significant reduction in TSC-RAML volume within the initial 6-month induction therapy period, and exhibited volume stabilization during the low-dose maintenance therapy treatment period without any instances of TSC-RAML regrowth. Adverse events (AEs) were significantly less severe and less frequent over the course of maintenance therapy relative to standard therapy. Conclusions: Low-dose everolimus maintenance therapy represents an effective approach to achieving TSC-RAML control following a minimum of 6 months of full-dose induction therapy, and may be associated with decreases in everolimus-related AE frequency and severity.
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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
影响因子：
3.7
作者：
Cai Y;Guo H;Wang W;Li H;Sun H;Shi B;Zhang Y
通讯作者：
Zhang Y
结节性硬化症复杂诊断标准更新：2012年iinternation Tuberous硬化症复杂共识会议的建议。
影响因子：
3.8
作者：
Northrup H;Krueger DA;International Tuberous Sclerosis Complex Consensus Group
通讯作者：
International Tuberous Sclerosis Complex Consensus Group
影响因子：
81.5
作者：
Henske, Elizabeth P.;Jozwiak, Sergiusz;Thiele, Elizabeth A.
通讯作者：
Thiele, Elizabeth A.
影响因子：
168.9
作者：
Bissler, John J.;Kingswood, J. Christopher;Budde, Klemens
通讯作者：
Budde, Klemens
影响因子：
3.2
作者：
Trelinska, Joanna;Dachowska, Iwona;Mlynarski, Wojciech
通讯作者：
Mlynarski, Wojciech