Takashi Hatano1, Taishi Matsu-Ura2, Kei-Ichiro Mori2, Hiroyuki Inaba3, Katsuhisa Endo2, Mayumi Tamari4, Shin Egawa3. 1. Department of Urology, JR Tokyo General Hospital, 2-1-3 Yoyogi Shibuya-ku, Tokyo, 151-8528, Japan. hatano-t@jreast.co.jp. 2. Department of Urology, JR Tokyo General Hospital, 2-1-3 Yoyogi Shibuya-ku, Tokyo, 151-8528, Japan. 3. Department of Urology, Jikei University School of Medicine, Tokyo, Japan. 4. Research Center for Medical Science Core Research Facilities for Basic Science, Jikei University School of Medicine, Tokyo, Japan.
Abstract
BACKGROUND: The aim of this study was to evaluate the effects and the utility of second-line everolimus treatment for regrown renal angiomyolipoma (AML) with tuberous sclerosis complex (TSC) after transcatheter arterial embolization (TAE). METHODS: We investigated a total of 14 patients who underwent second-line everolimus treatment for TSC-AML that regrew after TAE, and assessed their effects and adverse events. Everolimus treatment was performed for AML with a maximum diameter of 4 cm. To determine the reduction ratio of AML, the volume of AML was measured using multislice helical computed tomography. Adverse events were evaluated according to CTCAE v4.0-JCOG. We further compared the treatment effect and adverse events with those in patients receiving first-line everolimus treatment. RESULTS: The AML volume decreased in all patients, with a ≥ 50% volume decrease in 57% (8 of 14) of the cases, and the mean reduction rate was 53%. We observed no significant difference in the mean reduction rate of AML between second-line everolimus treatment for regrown TSC-AML after TAE and first-line everolimus treatment for TSC-AML. The adverse events were mild and consistent with those reported in our previous study. CONCLUSION: Although further studies are needed, everolimus appears to be effective as second-line treatment for TSC-AML that regrew after TAE and a beneficial treatment option for TSC-AML.
BACKGROUND: The aim of this study was to evaluate the effects and the utility of second-line everolimus treatment for regrown renal angiomyolipoma (AML) with tuberous sclerosis complex (TSC) after transcatheter arterial embolization (TAE). METHODS: We investigated a total of 14 patients who underwent second-line everolimus treatment for TSC-AML that regrew after TAE, and assessed their effects and adverse events. Everolimus treatment was performed for AML with a maximum diameter of 4 cm. To determine the reduction ratio of AML, the volume of AML was measured using multislice helical computed tomography. Adverse events were evaluated according to CTCAE v4.0-JCOG. We further compared the treatment effect and adverse events with those in patients receiving first-line everolimus treatment. RESULTS: The AML volume decreased in all patients, with a ≥ 50% volume decrease in 57% (8 of 14) of the cases, and the mean reduction rate was 53%. We observed no significant difference in the mean reduction rate of AML between second-line everolimus treatment for regrown TSC-AML after TAE and first-line everolimus treatment for TSC-AML. The adverse events were mild and consistent with those reported in our previous study. CONCLUSION: Although further studies are needed, everolimus appears to be effective as second-line treatment for TSC-AML that regrew after TAE and a beneficial treatment option for TSC-AML.
Authors: John J Bissler; John Christopher Kingswood; Elżbieta Radzikowska; Bernard A Zonnenberg; Michael Frost; Elena Belousova; Matthias Sauter; Norio Nonomura; Susanne Brakemeier; Petrus J de Vries; Noah Berkowitz; Sara Miao; Scott Segal; Severine Peyrard; Klemens Budde Journal: Nephrol Dial Transplant Date: 2015-07-08 Impact factor: 5.992