PMID- 34901059
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20211215
IS  - 2296-858X (Print)
IS  - 2296-858X (Electronic)
IS  - 2296-858X (Linking)
VI  - 8
DP  - 2021
TI  - Low-Dose Everolimus Maintenance Therapy for Renal Angiomyolipoma Associated With 
      Tuberous Sclerosis Complex.
PG  - 744050
LID - 10.3389/fmed.2021.744050 [doi]
LID - 744050
AB  - 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.
CI  - Copyright (c) 2021 Luo, Ye, Zu, Chen, Qi, Li and Cai.
FAU - Luo, Cong
AU  - Luo C
AD  - Department of Urology, National Clinical Research Center for Geriatric Disorders, 
      Xiangya Hospital, Central South University, Changsha City, China.
FAU - Ye, Wen-Rui
AU  - Ye WR
AD  - Department of Neurosurgery, Xiangya Hospital, Central South University, Changsha 
      City, China.
FAU - Zu, Xiong-Bin
AU  - Zu XB
AD  - Department of Urology, National Clinical Research Center for Geriatric Disorders, 
      Xiangya Hospital, Central South University, Changsha City, China.
FAU - Chen, Min-Feng
AU  - Chen MF
AD  - Department of Urology, National Clinical Research Center for Geriatric Disorders, 
      Xiangya Hospital, Central South University, Changsha City, China.
FAU - Qi, Lin
AU  - Qi L
AD  - Department of Urology, National Clinical Research Center for Geriatric Disorders, 
      Xiangya Hospital, Central South University, Changsha City, China.
FAU - Li, Yang-Le
AU  - Li YL
AD  - Department of Urology, National Clinical Research Center for Geriatric Disorders, 
      Xiangya Hospital, Central South University, Changsha City, China.
FAU - Cai, Yi
AU  - Cai Y
AD  - Department of Urology, National Clinical Research Center for Geriatric Disorders, 
      Xiangya Hospital, Central South University, Changsha City, China.
LA  - eng
PT  - Journal Article
DEP - 20211124
PL  - Switzerland
TA  - Front Med (Lausanne)
JT  - Frontiers in medicine
JID - 101648047
PMC - PMC8652067
OTO - NOTNLM
OT  - everolimus
OT  - low-dose maintenance therapy
OT  - renal angiomyolipoma
OT  - safety
OT  - tuberous sclerosis complex
COIS- The authors declare that the research was conducted in the absence of any 
      commercial or financial relationships that could be construed as a potential 
      conflict of interest.
EDAT- 2021/12/14 06:00
MHDA- 2021/12/14 06:01
PMCR- 2021/11/24
CRDT- 2021/12/13 18:09
PHST- 2021/07/19 00:00 [received]
PHST- 2021/11/05 00:00 [accepted]
PHST- 2021/12/13 18:09 [entrez]
PHST- 2021/12/14 06:00 [pubmed]
PHST- 2021/12/14 06:01 [medline]
PHST- 2021/11/24 00:00 [pmc-release]
AID - 10.3389/fmed.2021.744050 [doi]
PST - epublish
SO  - Front Med (Lausanne). 2021 Nov 24;8:744050. doi: 10.3389/fmed.2021.744050. 
      eCollection 2021.