Jeng-Dau Tsai1,2, Chang-Ching Wei3,4, Sheng-Hui Yang5, Hueng-Chuen Fan6, Chih-Chuan Hsu6, Min-Che Tung7, Min-Ling Tsai2,8, Ji-Nan Sheu1,2. 1. Department of Paediatrics, Chung Shan Medical University Hospital, Taichung, Taiwan. 2. School of Medicine, Chung Shan Medical University, Taichung, Taiwan. 3. Children's Hospital, China Medical University Hospital, Taichung, Taiwan. 4. School of Medicine, China Medical University, Taichung, Taiwan. 5. Department of Life Sciences, National Chung Hsing University, Taichung, Taiwan. 6. Departments of Paediatrics, Tungs' Taichung MetroHarbor Hospital, Taichung, Taiwan. 7. Departments of Surgery, Tungs' Taichung MetroHarbor Hospital, Taichung, Taiwan. 8. Department of Pharmacy, Chung Shan Medical University Hospital, Taichung, Taiwan.
Abstract
AIM: 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. CONCLUSION: 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.
AIM: 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. CONCLUSION: 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: Raouf M Seyam; Waleed K Alkhudair; Said A Kattan; Mohamed F Alotaibi; Hassan M Alzahrani; Waleed M Altaweel Journal: J Kidney Cancer VHL Date: 2017-10-16