mTOR標靶治療及動脈栓塞治療對於多發性硬化症合併腎臟血管平滑肌脂肪瘤之治療：病例系列報告
Mammalian Target of Rapamycin Inhibitor and Transarterial Embolization of Tuberous Sclerosis Complex-associated Renal Angiomyolipoma: A Case Series
蕭惟中1、蔡政道2、高育琳1, 3、王紹全1、陳文榮1、謝佐宜1、陳順郎1, 3
1中山醫學大學附設醫院泌尿科，2中山醫學大學附設醫院小兒科，3中山醫學大學
Wei-Chung Hsiao1, Jeng-Dau Tsai2, Yu-Lin Kao1, 3, Shao-Chuan Wang1 , Wen-Jung Chen1 , Tzuo-Yi Hsieh1 & Sung-Lang Chen1, 3
1Department of Urology, 2Department of Pediatrics, Chung Shan Medical University Hospital, Taichung, Taiwan, ROC
3School of Medicine, Chung Shan Medical University, Taichung, Taiwan, ROC
Abstract
Background: Renal angiomyolipomas (AML) are benign tumors composed of blood vessels, smooth muscle cells and fat cells. AML-related chronic kidney disease and tumor bleeding are the main cause of mortality. AML are highly associated with Tuberous Sclerosis Complex (TSC) and mostly be treated by mammalian target of rapamycin inhibitor (mTORi) therapy or transarterial embolization (TAE).
Materials and methods: Here we present our cases of TSC-associated renal AMLs treated with monotherapy or combined treatment of mTORi and TAE. A total of 19 patients (4 males and 15 females) were included. There were 10 patients treated by mTORi only and 9 by combined therapy with mTORi and TAE.
Results: The mean age of the patients was 34.5 (14-72) years. The maximal diameters of renal AMLs were from 1.5 to 20.9 (9.3±6.1) cm. During a mean follow-up duration of 43 (6-81) months, the size of AMLs decreased 15-33% in 8 patients receiving combination therapy. Two patients undergone monotherapy experienced 30-50% increase of tumor diameter. The tumor size of other patients showed no statistically significant change.
Conclusions: TSC-associated AMLs are quite common in this hereditary, complex syndrome. They are usually multiple and bilateral. The lesion usually progress over time and complicated with flank pain, hemorrhage, and even renal deterioration. TSC-associated AMLs can be treated by medication or minimally invasive therapy. Our result discovered although the tumor size may not shrink, TAE still play an important role in management of tumor bleeding with acceptable complications. In our study, combination therapy showed better control of tumor size than monotherapy alone in TSC-associated AMLs.