预测结节性硬化症患者肾血管平滑肌脂肪瘤接受 mTOR 抑制剂治疗效果的新分期标准。
New staging criteria predicting m-tor inhibitors treatment effect of renal angiomyolipoma in tuberous sclerosis complex patients.
机构信息
Department of Urology, Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, 1 Shuaifuyuan Wangfujing, Dongcheng, Beijing, 100730, PR China.
Department of Urologic Sciences, Vancouver Prostate Centre, University of British Columbia, Vancouver, BC, V6H 3Z6, Canada.
出版信息
World J Urol. 2024 Sep 20;42(1):532. doi: 10.1007/s00345-024-05235-3.
BACKGROUND
We aimed to launched new staging criteria to predict mTOR inhibitors treatment effect of renal angiomyolipomas (r-AMLs) in TSC patients.
METHODS
40 TSC patients with 69 r-AMLs were divided into two groups based on the efficacy of 6-month mTOR inhibitor treatment. Epidemiological data, therapeutic response, and predictive factors of enrolled patients were collected and analyzed. Age, sex, maximum diameter, maximum cross-sectional area (CSAmax), unenhanced mean CT value, enhanced mean CT value, and added value of enhanced CT of largest r-AML at baseline were assessed as potential influencing factors. Receiver operating characteristic (ROC) curve analysis and the area under the ROC curve (AUC) was used to estimate prediction power.
RESULTS
After 6 months of mTOR inhibitor treatment, the tumor reduction rates in the two groups were 55.87% and 16.44% (P < 0.001). At the start of treatment, the maximum diameters, CSAmax, added value of enhanced CT of the target lesion in two groups were 7.70 ± 0.73 cm vs. 13.18 ± 1.23 cm(P = 0.028), 57.40 ± 10.76cm2 vs. 167.29 ± 33.09cm2 (P = 0.015), and 62.32 ± 5.03HU vs. 33.06 ± 3.13HU (P = 0.009), respectively. AUCs of CSAmax, added value of enhanced CT, and combination of both were 0.8024, 0.7672, and 0.8116, respectively (P < 0.001). Cut-off values of CSAmax combined with the added value of enhanced CT were 40cm2 and 46HU. AUCs of maximum diameters, combination of maximum diameters and added value of enhanced CT were 0.7600 and 0.8100, respectively (P < 0.001), with cut-off values of 6.6 cm and 46 HU.
CONCLUSION
New staging criteria, based on CSAmax and added value of enhanced CT, can predict the treatment efficiency of m-TOR inhibitors for r-AMLs in TSC patients. A simplified version based on maximum diameter and added value of enhanced CT of lesion has also been proposed.
背景
我们旨在提出新的分期标准，以预测 TSC 患者肾血管平滑肌脂肪瘤（r-AMLs）接受 mTOR 抑制剂治疗的效果。
方法
根据 6 个月 mTOR 抑制剂治疗的疗效，将 40 名 TSC 患者的 69 个 r-AMLs 分为两组。收集并分析入组患者的流行病学数据、治疗反应和预测因素。评估基线时最大直径、最大横截面积（CSAmax）、增强 CT 平均值、增强 CT 增加值和最大 r-AML 的增强 CT 的附加值作为潜在影响因素。使用受试者工作特征（ROC）曲线分析和 ROC 曲线下面积（AUC）来估计预测能力。
结果
在接受 mTOR 抑制剂治疗 6 个月后，两组的肿瘤缩小率分别为 55.87%和 16.44%（P<0.001）。在治疗开始时，两组的目标病变最大直径、CSAmax 和增强 CT 增加值分别为 7.70±0.73cm 与 13.18±1.23cm（P=0.028）、57.40±10.76cm2 与 167.29±33.09cm2（P=0.015）和 62.32±5.03HU 与 33.06±3.13HU（P=0.009）。CSAmax、增强 CT 增加值和两者结合的 AUC 分别为 0.8024、0.7672 和 0.8116（P<0.001）。CSAmax 与增强 CT 增加值结合的截断值分别为 40cm2 和 46HU。最大直径、最大直径与增强 CT 增加值结合的 AUC 分别为 0.7600 和 0.8100（P<0.001），截断值分别为 6.6cm 和 46HU。
结论
基于 CSAmax 和增强 CT 增加值的新分期标准可预测 TSC 患者 r-AMLs 接受 mTOR 抑制剂治疗的疗效。还提出了一种基于病变最大直径和增强 CT 增加值的简化版本。
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