title: Effects of everolimus on tuberous sclerosis complex-associated renal angiomyolipoma: A preliminary report.
abstract: OBJECTIVE
  Tuberous sclerosis complex (TSC) presents with multisystem benign neoplasm induced by dysregulation of the mammalian target of rapamycin pathway. This study aimed to examine the effects of oral everolimus at either 2.5 or 5.0 mg daily on the treatment of TSC-associated renal angiomyolipoma (AML).
  
  
  METHODS
  Between July 2012 and August 2015, patients with TSC-associated renal AML were selected for everolimus therapy protocol. An oral everolimus starting dose at 2.5 mg was administered daily, and was gradually increased to 5.0 mg daily. All patients were evaluated using magnetic resonance imaging or computed tomography scanning at baseline, 12, 24, and 36 months after the start of treatment for measuring the changes of renal AML mass volume.
  
  
  RESULTS
  Eight patients were finally enrolled for analysis in this study. Everolimus treatment had a statistically significant effect on the renal AML volume reduction during follow-up (P < 0.05). Renal AML mass volume reduction rates were 10.5-45.3% in four patients with everolimus 2.5 mg and 40.7-73.1% in four patients with everolimus 5.0 mg daily; the difference was statistically significant between the two groups (P < 0.05). Longitudinal follow-up for response to everolimus showed volume reduction rates to be around 10.5-73.1% in the initial 6-24 months after everolimus treatment, which remained stable during follow-up up to 36 months.
  
  
  CONCLUSIONS
  The results suggest that an oral everolimus is effective and provides a non-invasive way to treat TSC-associated renal AML, and patients are likely to require maintenance therapy to continue to derive benefit.
authors: Department of Paediatrics, Chung Shan Medical University Hospital, Taichung, Taiwan.|School of Medicine, Chung Shan Medical University, Taichung, Taiwan.
authors: Jeng-Dau
authors: JD
authors: Tsai
authors: Children's Hospital, China Medical University Hospital, Taichung, Taiwan.|School of Medicine, China Medical University, Taichung, Taiwan.
authors: Chang-Ching
authors: CC
authors: Wei
authors: Department of Life Sciences, National Chung Hsing University, Taichung, Taiwan.
authors: Sheng-Hui
authors: SH
authors: Yang
authors: Departments of Paediatrics, Tungs' Taichung MetroHarbor Hospital, Taichung, Taiwan.
authors: Hueng-Chuen
authors: HC
authors: Fan
authors: Departments of Paediatrics, Tungs' Taichung MetroHarbor Hospital, Taichung, Taiwan.
authors: Chih-Chuan
authors: CC
authors: Hsu
authors: Departments of Surgery, Tungs' Taichung MetroHarbor Hospital, Taichung, Taiwan.
authors: Min-Che
authors: MC
authors: Tung
authors: School of Medicine, Chung Shan Medical University, Taichung, Taiwan.|Department of Pharmacy, Chung Shan Medical University Hospital, Taichung, Taiwan.
authors: Min-Ling
authors: ML
authors: Tsai
authors: Department of Paediatrics, Chung Shan Medical University Hospital, Taichung, Taiwan.|School of Medicine, Chung Shan Medical University, Taichung, Taiwan.
authors: Ji-Nan
authors: JN
authors: Sheu
mesh_terms: D000293:Adolescent; D000328:Adult; D018207:Angiomyolipoma / Q000188:drug therapy*; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D000068338:Everolimus / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007680:Kidney Neoplasms / Q000188:drug therapy*; D008297:Male; D014402:Tuberous Sclerosis / Q000150:complications*
chemical_list: D000970:Antineoplastic Agents; D000068338:Everolimus
keywords: everolimus; mass volume reduction; renal angiomyolipomas; the mammalian target of rapamycin; tuberous sclerosis complex