Abstract
The aim of this study was to evaluate the safety and efficacy of low-dose everolimus treatment in patients with tuberous sclerosis complex (TSC)-associated angiomyolipoma (AML) with renal dysfunction or low body weight. We investigated a total of 50 adult patients underwent everolimus treatment for AML associated with TSC. For patients with renal dysfunction (serum creatinine level ≥ 1.5mg/dl) or low body weight (body weight < 35kg), 5mg of everolimus was administered daily (low-dose group). For patients without renal dysfunction or low body weight, 10mg of everolimus was administered daily (conventional-dose group). The treatment effects and adverse events were compared between the two groups. There were 20 patients in the low-dose group, and 30 in the conventional-dose group. The average reduction rate of the AML volume in the low-dose group was 52%, whereas it was 60% in the conventional-dose group. No significant differences were found in the average reduction rate between the groups (P = 0.24). The average blood everolimus trough levels were 7.7 ± 3.1ng/mL in the low-dose group and 12.2 ± 5.7ng/mL in the conventional-dose group. The level was significantly higher in the conventional-dose group than in the low-dose group (P = 0.004). The incidences of stomatitis and irregular menstruation were significantly lower in the low-dose group than in the conventional-dose group (P = 0.009, P = 0.045, respectively). The present study demonstrates that low-dose everolimus treatment is safe and effective for TSC-associated AML. This treatment was well tolerated and adverse events were mild in all cases.
Highlights
Tuberous sclerosis complex (TSC) is a rare disease, with an autosomal dominant mode of inheritance, that results in systemic hamartoma
This treatment was well tolerated and adverse events were mild in all cases
Tuberous sclerosis complex (TSC) is a systemic disease with an autosomal dominant mode of inheritance, in which hamartomas form throughout the body, leading to the development of various organ disorders[1]
Summary
Tuberous sclerosis complex (TSC) is a rare disease, with an autosomal dominant mode of inheritance, that results in systemic hamartoma. Renal angiomyolipoma (AML) is observed in 50-80% of TSC patients. Everolimus is recommended as an initial treatment for TSC-associated AML; few studies have investigated the efficacy and safety of low-dose everolimus therapy. We evaluated the safety and efficacy of low-dose everolimus treatment in patients with TSC-associated AML with renal dysfunction or low body weight. Tuberous sclerosis complex (TSC) is a systemic disease with an autosomal dominant mode of inheritance, in which hamartomas form throughout the body, leading to the development of various organ disorders[1]. AML is the most frequent benign renal tumor and is observed in 50%-80% of TSC patients[4]. When the tumor size is ≥4 cm, the growing speed is fast, and the incidence of hemorrhaging and hematuria increases[5]
Sign-in/Register to view highlights & summary Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
