Tumor fat composition predicts response to everolimus and supports low-dose therapy in TSC-associated renal angiomyolipomas: a multicenter prospective study

Gao K, Zhang X et al. · 2026 · Frontiers in oncology · Atlas ID GAO2026B

What this study shows

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. No significant difference in response was detected between the low-dose and standard-dose groups in an exploratory, non-randomised subset, and oral mucositis was less frequent on the low dose; tumor regrowth occurred after treatment discontinuation.

Abstract

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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.

Read the full abstract on PubMed →

At a glance

Evidence type	H Human study Marked H because it is direct evidence from a human clinical trial or human cohort; the code names the kind of study, not its quality -- a small, well-run trial is still H.
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
Record last updated	2026-10-01
Source	DOI 10.3389/fonc.2026.1893155 · PMID 42558341 · Free full text (PMC13437317)

Extracted findings

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; no significant difference between low and standard dose in an exploratory, non-randomised subset, with less oral mucositis on the low dose

In the Atlas

Answers that reference this study

Discussed in the plain-language answer How does mTOR connect to cancer?.

Cite this paper

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Gao, K., et al. (2026). Tumor fat composition predicts response to everolimus and supports low-dose therapy in TSC-associated renal angiomyolipomas: a multicenter prospective study. Frontiers in oncology. https://doi.org/10.3389/fonc.2026.1893155

@article{GAO2026B,
  author       = {Gao, K. and Zhang, X. and others},
  title        = {{Tumor fat composition predicts response to everolimus and supports low-dose therapy in TSC-associated renal angiomyolipomas: a multicenter prospective study}},
  journal      = {Frontiers in oncology},
  year         = {2026},
  doi          = {10.3389/fonc.2026.1893155},
  note         = {PMID: 42558341},
}

Cite this Atlas record

The record is the Atlas's own work — the evidence label, the extracted findings and the links. It is cited as part of the dataset, not as the paper.

Barton, O. (2026). Oliver's mTOR Atlas (record GAO2026B) [Data set]. https://mtor-atlas.org/study/GAO2026B/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_GAO2026B,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record GAO2026B},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/GAO2026B/},
  doi          = {10.5281/zenodo.22059963}
}

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