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Effects of everolimus on tuberous sclerosis complex-associated renal angiomyolipoma: A preliminary report
Jeng-Dau Tsai1,2, Chang-Ching Wei3,4, Sheng-Hui Yang5
1Department of Paediatrics, Chung Shan Medical University Hospital, Taichung, Taiwan.
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.
Insights
Oral everolimus effectively reduces renal angiomyolipoma (AML) volume in patients with tuberous sclerosis complex (TSC). Maintenance therapy may be needed for sustained benefits in treating TSC-associated AML.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- Tuberous sclerosis complex (TSC) is a genetic disorder characterized by benign neoplasms due to mTOR pathway dysregulation.
- Renal angiomyolipomas (AML) are common manifestations of TSC, potentially leading to significant complications.
Purpose of the Study:
- To evaluate the efficacy of oral everolimus in treating TSC-associated renal AML.
- To compare the effects of different daily doses (2.5 mg vs. 5.0 mg) of everolimus on renal AML volume reduction.
Main Methods:
- A prospective study involving eight patients with TSC-associated renal AML treated with oral everolimus.
- Dosing started at 2.5 mg daily, with potential escalation to 5.0 mg daily.
- Renal AML volume was assessed using MRI or CT scans at baseline and at 12, 24, and 36 months.
Main Results:
- Everolimus treatment resulted in a statistically significant reduction in renal AML volume.
- Patients receiving 5.0 mg daily showed greater volume reduction (40.7-73.1%) compared to those on 2.5 mg (10.5-45.3%).
- Volume reductions were observed within 6-24 months and remained stable up to 36 months.
Conclusions:
- Oral everolimus is an effective, non-invasive treatment for TSC-associated renal AML.
- Sustained benefits suggest the potential need for long-term maintenance therapy with everolimus.
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