連續低劑量癌伏妥在節結性硬化症合併腎臟血管肌肉脂肪瘤病患之療效-48個月追蹤研究
趙浩堅1、蔡政道2,3、王紹全1,3、陳順郎1,3
中山醫學大學附設醫院 1泌尿科 2小兒科
3中山醫學大學醫學系
Continuous low dose everolimus shrinkage tuberous sclerosis complex associated renal angiomyolipoma- a 48-month follow-up study
Hao-Chien Chao 1, Jeng-Dau Tsai2,3, Shao- Chuan Wang1.3 ,Sung-Lang Chen1,3,
1Department of Urology, 2Department of Paediatrics, Chung Shan Medical University Hospital, Taichung, Taiwan
3School of Medicine, Chung Shan Medical University, Taichung, Taiwan
Purpose: Tuberous sclerosis complex (TSC) is a rare disease that causes multisystem benign neoplasm, induced by dysregulation of the mammalian target of the rapamycin pathway. This study aimed to examine the effects of low-dose everolimus, a potent and selective inhibitor of mTOR, on the treatment of TSC-associated renal angiomyolipoma (AML).
Materials and Methods: Between July 2013 and August 2017, 11 patients with TSC-AML were enrolled for an everolimus therapy protocol. An oral everolimus dose starting at 2.5 mg daily, was gradually increased to 5.0 mg daily. All patients were evaluated using magnetic resonance imaging or computed tomography scanning at baseline, 12, 24, 36 and 48 months after the start of treatment for measuring changes of renal AML mass volume.
Results: Everolimus therapy resulted in significant shrinkage of TSC-AML volume after 48 months follow-up. Serum levels of everolimus were subdivided into group I (<8 ng/ ml, n=6), and group II (>8 ng/ ml, n=5). The volume reduction rates were 10.6% to 65.2 % in group I and 42.5% to 70.6% in group II. To evaluate the response to treatment, 3 of six (50%) were responders in group I, and all the patients in group II (5/5, 100%) were responders. The differences in AML volume reduction between the groups were statistically significant at 12 months (p=0.011), 24 months (p=0,006), 36 months (p=0.014) and 48 months (p=0.05).
Conclusion: These results suggest that continuous low-dose everolimus therapy is effective in shrinking TSC-AML volume, and minimizes adverse effects and subsequent reducing medical costs.