Objectives: To assess the efficacy and safety of preoperative neoadjuvant everolimus in renal angiomyolipomas (AML) patients with or without Tuberous Sclerosis Complex (TSC). Materials and methods: This multi-institutional retrospective study enrolled renal AML patients who underwent partial nephrectomy (PN) or total nephrectomy after receiving at least 1 month of pre-operative everolimus. Imaging evaluations were collected before and after treatment, along with demographic, surgical, and follow-up information. The primary outcome was tumor volume reduction of >= 25%, with additional outcomes including recurrence, perioperative outcomes, renal function, and safety. Results: From January 2015 to July 2022, 68 renal AML patients were studied-41 with TSC and 27 without. During everolimus treatment, 61.0% (25/41) of TSC patients and 44.4% (12/27) of non-TSC patients achieved tumor reduction of >= 25%. Additionally, 41.5% (17/41) of TSC patients and 18.5% (5/27) of non-TSC patients achieved a >= 50% reduction. Three TSC patients and 1 non-TSC patient discontinued treatment due to side-effects. Most patients (92.7% TSC, 85.2% non-TSC) underwent PN. After everolimus treatment, the necessary total nephrectomy decreased to 41.2% (7/17) from baseline. Postoperatively, 1 grade 3 and 3 grade 2 complications occurred, with no grade 4 or 5 complications. After a median follow-up of 24 months, only 1 TSC patient recurred with a diameter >3 cm. Retrospective nature is the major limitation of this study. Conclusion: Everolimus was effective and well-tolerated in neoadjuvant treatment for renal AML, especially in TSC patients. This neoadjuvant combination strategy of everolimus and PN could effectively controls recurrence and preserves renal function.
基金：
Shanghai Science and Technology
Commission Research Project, Grant/
Award Number: 21ZR1438900; the
Incubating Program for Clinical
Research and Innovation of Renji
Hospital, Grant/Award Number:
PYIII20-07 and PYXJS16-008; Wu
Jieping Medical Foundation, Grant/
Award Number: 320.6750.2022-19-92;
Basic Oncology Research Program
from Bethune Charitable Foundation,
Grant/Award Number: BCF-NH-ZL20201119-024
第一作者机构：[1]Shanghai Jiao Tong Univ, Ren Ji Hosp, Sch Med, Dept Urol, 1630 Dongfang Rd, Shanghai 200127, Peoples R China
共同第一作者：
通讯作者：
通讯机构：[1]Shanghai Jiao Tong Univ, Ren Ji Hosp, Sch Med, Dept Urol, 1630 Dongfang Rd, Shanghai 200127, Peoples R China[2]Sun Yat Sen Univ Canc Ctr, Collaborat Innovat Ctr Canc Med, Dept Urol Oncol, State Key Lab Oncol South China, 651 Dongfeng East Rd, Guangzhou 510060, Peoples R China[3]Chinese Peoples Liberat Army Gen Hosp, Med Ctr 3, Dept Urol, Beijing, Peoples R China[*1]Department of Urology, Ren Ji Hospital, School of Medicine, Shanghai Jiao Tong University, 1630 Dongfang Road, Pudong District, Shanghai 200127, China.[*2]Department of Urology Oncology, Sun Yat-sen University Cancer Center ,State Key Laboratory of Oncology in South China, Collaborative Innovation Center of Cancer Medicine, 651 Dongfeng East Road, Yuexiu District, Guangzhou, 510060, China[*3]Department of Urology, the Third Medical Centre, Chinese PLA General Hospital, Beijing, China
推荐引用方式(GB/T 7714)：
Su Ruopeng,Huang Tingxuan,Gu Liangyou,et al.Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study[J].CANCER MEDICINE.2024,13(17):doi:10.1002/cam4.70181.
APA：
Su, Ruopeng,Huang, Tingxuan,Gu, Liangyou,Bao, Yige,Liu, Zhihong...&Zhang, Jin.(2024).Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study.CANCER MEDICINE,13,(17)
MLA：
Su, Ruopeng,et al."Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study".CANCER MEDICINE 13..17(2024)
