Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study

Abstract 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...

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Main Authors: Ruopeng Su, Tingxuan Huang, Liangyou Gu, Yige Bao, Zhihong Liu, Pinghong Dao, Lin Yao, Xiaoyi Hu, Guanghou Fu, Jitao Wu, Thibault Tricard, Guangyu Wu, Minfeng Chen, Chancan Li, Zhiyang Huang, Bing Zheng, Yonghui Chen, Wei Xue, Gang Guo, Pei Dong, Jiwei Huang, Jin Zhang
Format: Article
Language:English
Published: Wiley 2024-09-01
Series:Cancer Medicine
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Online Access:https://doi.org/10.1002/cam4.70181
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author Ruopeng Su
Tingxuan Huang
Liangyou Gu
Yige Bao
Zhihong Liu
Pinghong Dao
Lin Yao
Xiaoyi Hu
Guanghou Fu
Jitao Wu
Thibault Tricard
Guangyu Wu
Minfeng Chen
Chancan Li
Zhiyang Huang
Bing Zheng
Yonghui Chen
Wei Xue
Gang Guo
Pei Dong
Jiwei Huang
Jin Zhang
author_facet Ruopeng Su
Tingxuan Huang
Liangyou Gu
Yige Bao
Zhihong Liu
Pinghong Dao
Lin Yao
Xiaoyi Hu
Guanghou Fu
Jitao Wu
Thibault Tricard
Guangyu Wu
Minfeng Chen
Chancan Li
Zhiyang Huang
Bing Zheng
Yonghui Chen
Wei Xue
Gang Guo
Pei Dong
Jiwei Huang
Jin Zhang
author_sort Ruopeng Su
collection DOAJ
description Abstract 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.
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spelling doaj-art-d72d1762ebea4d768e0a85bafd895d032025-02-07T09:08:08ZengWileyCancer Medicine2045-76342024-09-011317n/an/a10.1002/cam4.70181Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective studyRuopeng Su0Tingxuan Huang1Liangyou Gu2Yige Bao3Zhihong Liu4Pinghong Dao5Lin Yao6Xiaoyi Hu7Guanghou Fu8Jitao Wu9Thibault Tricard10Guangyu Wu11Minfeng Chen12Chancan Li13Zhiyang Huang14Bing Zheng15Yonghui Chen16Wei Xue17Gang Guo18Pei Dong19Jiwei Huang20Jin Zhang21Department of Urology Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine Shanghai ChinaDepartment of Urology Oncology Sun Yat‐sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center of Cancer Medicine Guangzhou ChinaDepartment of Urology, The Third Medical Centre Chinese PLA General Hospital Beijing ChinaDepartment of Urology and Institute of Urology West China Hospital, Sichuan University Chengdu ChinaDepartment of Urology and Institute of Urology West China Hospital, Sichuan University Chengdu ChinaDepartment of Urology Xiangya Hospital, Central South University Changsha ChinaDepartment of Urology First Hospital of Peking University, Institute of Urology, Peking University, National Urological Cancer Center Beijing ChinaDepartment of Urology Zhongshan Hospital, Fudan University Shanghai ChinaDepartment of Urology The First Affiliated Hospital, School of Medicine, Zhejiang University Hangzhou ChinaDepartment of Urology Yantai Yuhuangding Hospital, Qingdao University Yantai Shandong ChinaDepartment of Urology Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine Shanghai ChinaDepartment of Radiology Renji Hospital, Shanghai Jiao Tong University School of Medicine Shanghai ChinaDepartment of Urology Xiangya Hospital, Central South University Changsha ChinaThe Department of Urology AnHui NO.2 Provincial People Hospital Hefei ChinaDepartment of Urology Quanzhou First Hospital affiliated to Fujian Medical University Quanzhou ChinaThe Department of Urology The Second Affiliated Hospital of Nantong University Nantong ChinaDepartment of Urology Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine Shanghai ChinaDepartment of Urology Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine Shanghai ChinaDepartment of Urology, The Third Medical Centre Chinese PLA General Hospital Beijing ChinaDepartment of Urology Oncology Sun Yat‐sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center of Cancer Medicine Guangzhou ChinaDepartment of Urology Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine Shanghai ChinaDepartment of Urology Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine Shanghai ChinaAbstract 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.https://doi.org/10.1002/cam4.70181adverse eventseverolimusneoadjuvantrenal angiomyolipomastreatment responsetuberous sclerosis complex
spellingShingle Ruopeng Su
Tingxuan Huang
Liangyou Gu
Yige Bao
Zhihong Liu
Pinghong Dao
Lin Yao
Xiaoyi Hu
Guanghou Fu
Jitao Wu
Thibault Tricard
Guangyu Wu
Minfeng Chen
Chancan Li
Zhiyang Huang
Bing Zheng
Yonghui Chen
Wei Xue
Gang Guo
Pei Dong
Jiwei Huang
Jin Zhang
Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study
Cancer Medicine
adverse events
everolimus
neoadjuvant
renal angiomyolipomas
treatment response
tuberous sclerosis complex
title Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study
title_full Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study
title_fullStr Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study
title_full_unstemmed Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study
title_short Neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex: Results from a multicenter, retrospective study
title_sort neoadjuvant everolimus in renal angiomyolipoma with or without tuberous sclerosis complex results from a multicenter retrospective study
topic adverse events
everolimus
neoadjuvant
renal angiomyolipomas
treatment response
tuberous sclerosis complex
url https://doi.org/10.1002/cam4.70181
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