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

IF 2.9 2区医学 Q2 ONCOLOGY Cancer Medicine Pub Date : 2024-09-15 DOI:10.1002/cam4.70181

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

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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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新辅助依维莫司治疗伴有或不伴有结节性硬化综合征的肾血管肌脂肪瘤：一项多中心回顾性研究的结果

目的评估依维莫司在伴有或不伴有结节性硬化综合征（TSC）的肾血管脂肪瘤（AML）患者术前新辅助治疗的有效性和安全性。材料与方法这项多机构回顾性研究纳入了接受肾部分切除术（PN）或全肾切除术的肾脏AML患者，他们在术前接受了至少1个月的依维莫司治疗。研究人员收集了治疗前后的影像学评估以及人口统计学、手术和随访信息。主要结果是肿瘤体积缩小≥25%，其他结果包括复发、围手术期结果、肾功能和安全性。结果从2015年1月到2022年7月，共研究了68例肾性AML患者--41例有TSC，27例无TSC。在依维莫司治疗期间，61.0%（25/41）的TSC患者和44.4%（12/27）的非TSC患者的肿瘤缩小≥25%。此外，41.5%（17/41）的 TSC 患者和 18.5%（5/27）的非 TSC 患者的肿瘤缩小率≥50%。3名TSC患者和1名非TSC患者因副作用停止了治疗。大多数患者（92.7% 的 TSC 患者和 85.2% 的非 TSC 患者）接受了 PN 治疗。依维莫司治疗后，必要的全肾切除率从基线降至 41.2%（7/17）。术后发生了1例3级和3例2级并发症，无4级或5级并发症。中位随访24个月后，仅有1例TSC患者复发，直径达3厘米。回顾性是本研究的主要局限性。结论依维莫司在肾性急性髓细胞白血病的新辅助治疗中疗效显著，耐受性良好，尤其适用于TSC患者。依维莫司和PN的新辅助联合治疗策略可有效控制复发并保护肾功能。

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来源期刊

Cancer Medicine ONCOLOGY-

CiteScore

5.50

自引率

2.50%

发文量

907

审稿时长

19 weeks

期刊介绍： Cancer Medicine is a peer-reviewed, open access, interdisciplinary journal providing rapid publication of research from global biomedical researchers across the cancer sciences. The journal will consider submissions from all oncologic specialties, including, but not limited to, the following areas: Clinical Cancer Research Translational research ∙ clinical trials ∙ chemotherapy ∙ radiation therapy ∙ surgical therapy ∙ clinical observations ∙ clinical guidelines ∙ genetic consultation ∙ ethical considerations Cancer Biology: Molecular biology ∙ cellular biology ∙ molecular genetics ∙ genomics ∙ immunology ∙ epigenetics ∙ metabolic studies ∙ proteomics ∙ cytopathology ∙ carcinogenesis ∙ drug discovery and delivery. Cancer Prevention: Behavioral science ∙ psychosocial studies ∙ screening ∙ nutrition ∙ epidemiology and prevention ∙ community outreach. Bioinformatics: Gene expressions profiles ∙ gene regulation networks ∙ genome bioinformatics ∙ pathwayanalysis ∙ prognostic biomarkers. Cancer Medicine publishes original research articles, systematic reviews, meta-analyses, and research methods papers, along with invited editorials and commentaries. Original research papers must report well-conducted research with conclusions supported by the data presented in the paper.