Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex

Objective: To assess the safety and efficacy of low-dose everolimus maintenance therapy for tuberous sclerosis complex-related renal angiomyolipoma (TSC-RAML) patients that had previously undergone standard-dose treatment for a minimum of 6 months.Materials and Methods: In total, 24 patients with a...

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Autores principales:	Cong Luo, Wen-Rui Ye, Xiong-Bin Zu, Min-Feng Chen, Lin Qi, Yang-Le Li, Yi Cai
Formato:	article
Lenguaje:	EN
Publicado:	Frontiers Media S.A. 2021
Materias:	tuberous sclerosis complex renal angiomyolipoma everolimus low-dose maintenance therapy safety Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/ab88bfc009544f3fbb563fa102839896
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spelling	oai:doaj.org-article:ab88bfc009544f3fbb563fa1028398962021-11-30T18:11:07ZLow-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex2296-858X10.3389/fmed.2021.744050https://doaj.org/article/ab88bfc009544f3fbb563fa1028398962021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fmed.2021.744050/fullhttps://doaj.org/toc/2296-858XObjective: To assess the safety and efficacy of low-dose everolimus maintenance therapy for tuberous sclerosis complex-related renal angiomyolipoma (TSC-RAML) patients that had previously undergone standard-dose treatment for a minimum of 6 months.Materials and Methods: In total, 24 patients with a definitive TSC diagnosis were enrolled from April 2018 – April 2019 at Xiangya Hospital, Central South University. All patients underwent low-dose everolimus maintenance therapy following standard-dose everolimus induction therapy for a minimum of 6 months. Patients additionally underwent TSC1/TSC2 genetic testing, And they were followed-up at 3, 6, 12, 18, and 24 months. The Response Evaluation Criteria in Solid Tumors (RECIST, version 1.1) criteria were used to monitor patient RAML responses, while adverse events (AEs) were assessed as per the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE, version 4.0). P < 0.05 was the significance level for all analyses, which were performed using SPSS 19.0.Results: TSC1/TSC2 gene mutations were present in all 24 patients, all of whom achieved a significant reduction in TSC-RAML volume within the initial 6-month induction therapy period, and exhibited volume stabilization during the low-dose maintenance therapy treatment period without any instances of TSC-RAML regrowth. Adverse events (AEs) were significantly less severe and less frequent over the course of maintenance therapy relative to standard therapy.Conclusions: Low-dose everolimus maintenance therapy represents an effective approach to achieving TSC-RAML control following a minimum of 6 months of full-dose induction therapy, and may be associated with decreases in everolimus-related AE frequency and severity.Cong LuoWen-Rui YeXiong-Bin ZuMin-Feng ChenLin QiYang-Le LiYi CaiFrontiers Media S.A.articletuberous sclerosis complexrenal angiomyolipomaeverolimuslow-dose maintenance therapysafetyMedicine (General)R5-920ENFrontiers in Medicine, Vol 8 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	tuberous sclerosis complex renal angiomyolipoma everolimus low-dose maintenance therapy safety Medicine (General) R5-920
spellingShingle	tuberous sclerosis complex renal angiomyolipoma everolimus low-dose maintenance therapy safety Medicine (General) R5-920 Cong Luo Wen-Rui Ye Xiong-Bin Zu Min-Feng Chen Lin Qi Yang-Le Li Yi Cai Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex
description	Objective: To assess the safety and efficacy of low-dose everolimus maintenance therapy for tuberous sclerosis complex-related renal angiomyolipoma (TSC-RAML) patients that had previously undergone standard-dose treatment for a minimum of 6 months.Materials and Methods: In total, 24 patients with a definitive TSC diagnosis were enrolled from April 2018 – April 2019 at Xiangya Hospital, Central South University. All patients underwent low-dose everolimus maintenance therapy following standard-dose everolimus induction therapy for a minimum of 6 months. Patients additionally underwent TSC1/TSC2 genetic testing, And they were followed-up at 3, 6, 12, 18, and 24 months. The Response Evaluation Criteria in Solid Tumors (RECIST, version 1.1) criteria were used to monitor patient RAML responses, while adverse events (AEs) were assessed as per the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE, version 4.0). P < 0.05 was the significance level for all analyses, which were performed using SPSS 19.0.Results: TSC1/TSC2 gene mutations were present in all 24 patients, all of whom achieved a significant reduction in TSC-RAML volume within the initial 6-month induction therapy period, and exhibited volume stabilization during the low-dose maintenance therapy treatment period without any instances of TSC-RAML regrowth. Adverse events (AEs) were significantly less severe and less frequent over the course of maintenance therapy relative to standard therapy.Conclusions: Low-dose everolimus maintenance therapy represents an effective approach to achieving TSC-RAML control following a minimum of 6 months of full-dose induction therapy, and may be associated with decreases in everolimus-related AE frequency and severity.
format	article
author	Cong Luo Wen-Rui Ye Xiong-Bin Zu Min-Feng Chen Lin Qi Yang-Le Li Yi Cai
author_facet	Cong Luo Wen-Rui Ye Xiong-Bin Zu Min-Feng Chen Lin Qi Yang-Le Li Yi Cai
author_sort	Cong Luo
title	Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex
title_short	Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex
title_full	Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex
title_fullStr	Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex
title_full_unstemmed	Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex
title_sort	low-dose everolimus maintenance therapy for renal angiomyolipoma associated with tuberous sclerosis complex
publisher	Frontiers Media S.A.
publishDate	2021
url	https://doaj.org/article/ab88bfc009544f3fbb563fa102839896
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Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex

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