Department of Urology, Seirei Yokohama Hospital, Kanagawa, Japan.
Department of Urology, JR Tokyo General Hospital, 2-1-3 Yoyogi Shibuya-ku, Tokyo, 151-8528, Japan.
Int J Clin Oncol. 2021 Jan;26(1):163-168. doi: 10.1007/s10147-020-01792-w. Epub 2020 Sep 29.
The aim of this study was to evaluate the safety and efficacy of low-dose everolimus treatment in patients with tuberous sclerosis complex (TSC)-associated angiomyolipoma (AML) with renal dysfunction or low body weight.
We investigated a total of 50 adult patients underwent everolimus treatment for AML associated with TSC. For patients with renal dysfunction (serum creatinine level ≥ 1.5 mg/dl) or low body weight (body weight < 35 kg), 5 mg of everolimus was administered daily (low-dose group). For patients without renal dysfunction or low body weight, 10 mg of everolimus was administered daily (conventional-dose group). The treatment effects and adverse events were compared between the two groups.
There were 20 patients in the low-dose group, and 30 in the conventional-dose group. The average reduction rate of the AML volume in the low-dose group was 52%, whereas it was 60% in the conventional-dose group. No significant differences were found in the average reduction rate between the groups (P = 0.24). The average blood everolimus trough levels were 7.7 ± 3.1 ng/mL in the low-dose group and 12.2 ± 5.7 ng/mL in the conventional-dose group. The level was significantly higher in the conventional-dose group than in the low-dose group (P = 0.004). The incidences of stomatitis and irregular menstruation were significantly lower in the low-dose group than in the conventional-dose group (P = 0.009, P = 0.045, respectively).
The present study demonstrates that low-dose everolimus treatment is safe and effective for TSC-associated AML. This treatment was well tolerated and adverse events were mild in all cases.
本研究旨在评估低剂量依维莫司治疗伴有肾功能不全或低体重的结节性硬化症（TSC）相关血管平滑肌脂肪瘤（AML）患者的安全性和有效性。
我们共调查了 50 例接受依维莫司治疗 TSC 相关 AML 的成年患者。对于肾功能不全（血清肌酐水平≥1.5mg/dl）或低体重（体重<35kg）的患者，每日给予 5mg 依维莫司（低剂量组）。对于无肾功能不全或低体重的患者，每日给予 10mg 依维莫司（常规剂量组）。比较两组的治疗效果和不良反应。
低剂量组有 20 例患者，常规剂量组有 30 例患者。低剂量组 AML 体积的平均缩小率为 52%，而常规剂量组为 60%。两组平均缩小率无显著差异（P=0.24）。低剂量组的平均血依维莫司谷浓度为 7.7±3.1ng/ml，常规剂量组为 12.2±5.7ng/ml。常规剂量组的水平明显高于低剂量组（P=0.004）。低剂量组的口腔炎和月经不规律发生率明显低于常规剂量组（P=0.009，P=0.045）。
本研究表明，低剂量依维莫司治疗 TSC 相关 AML 安全有效。所有患者均耐受良好，不良反应轻微。
