In a multicenter prospective cohort of 183 TSC-associated renal angiomyolipoma patients, everolimus reduced tumor volume by >=50% in 44% of patients at 3 months and 83% at 6 months; fat-poor lesions responded significantly better than fat-rich ones. A low-dose everolimus regimen showed comparable efficacy to standard dose with fewer adverse events (notably less oral mucositis), though tumor regrowth occurred after treatment discontinuation.
| Evidence tier | B Direct human evidence |
| Study type | 3 - Human Observational |
| Model system | Human (TSC-associated renal angiomyolipoma patients, multicenter prospective cohort, n=183) |
| Journal | Frontiers in oncology |
| Year | 2026 |
| Peer reviewed | Yes |
| Source | DOI 10.3389/fonc.2026.1893155 · PMID 42558341 · Free full text (PMC13437317) |
Renal angiomyolipomas associated with tuberous sclerosis complex (TSC-RAML) may lead to progressive renal damage and hemorrhage. Everolimus is recommended as first-line therapy, but predictors of treatment response and optimal dosing strategies remain unclear. In this multicenter prospective cohort study across multiple centers in China, 183 consecutive patients with TSC-RAML were enrolled, with three prespecified analytical subsets: standard-dose everolimus efficacy, CT-based tumor fat-composition analysis, and exploratory dose-comparison analysis. A >=50% reduction in tumor volume occurred in 44% (34/78) of patients at 3 months and 83% (60/72) at 6 months. Tumor reduction was significantly greater in fat-poor than fat-rich lesions. No significant difference in response was observed between standard- and low-dose groups. Oral mucositis was the most common adverse event but occurred less frequently in the low-dose group (P=.002). Tumor regrowth was observed after treatment discontinuation. Limitations include the nonrandomized design and open-label treatment administration.
| Intervention | Everolimus, standard-dose vs low-dose; CT-based tumor fat composition analysis |
| Target | mTOR (via everolimus) |
| Model | Human (TSC-RAML patients, n=183, multicenter China cohort) |
| Effect | Everolimus reduces TSC-associated renal angiomyolipoma tumor volume, more effectively in fat-poor lesions; low-dose regimen shows comparable efficacy with better tolerability |
