Renal angiomyolipoma

Disease · 2 studies in the Atlas

TSC-associated benign renal tumour; the measured endpoint of EXIST-2.

A benign kidney growth that is common in tuberous sclerosis.

Exists as its own node because EXIST-2 measured renal angiomyolipoma response (42% vs 0% on placebo) in patients with TSC or sporadic LAM. Lung disease was not the endpoint, so an arrow drawn at LAM would claim more than the trial showed.

In the pathway model

1 interaction in the curated mTOR pathway model. Each row is a claim with the evidence behind it; the evidence code is the kind of study, not its quality.

Interaction	Mechanism	Evidence	Studies
Everolimus inhibits Renal angiomyolipoma clinical-outcome · on the map table	In another trial, everolimus shrank kidney growths in patients with tuberous sclerosis or a related lung disease, working in 42% of patients versus 0% on placebo. EXIST-2 tested everolimus against renal angiomyolipoma in patients with tuberous sclerosis or sporadic LAM, and shrank those lesions by >=50% in 42% versus 0% on placebo. EXIST-2 tested everolimus against renal angiomyolipoma in patients with tuberous sclerosis or sporadic LAM, and shrank those lesions by >=50% in 42% versus 0% on placebo.	H	BIS2013, GAO2026B

Evidence at a glance

Evidence	What it means	Studies
H	Human study	2

Studies

Year	Evidence	Study
2026	H	Tumor fat composition predicts response to everolimus and supports low-dose therapy in TSC-associated renal angiomyolipomas: a multicenter prospective study GAO2026B 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.
2013	H	Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial BIS2013 The companion phase 3 RCT (n=118) to EXIST-1, targeting kidney tumors (angiomyolipomas) in tuberous sclerosis and LAM. Everolimus shrank them by >=50% in 42% of patients versus 0% on placebo. Together EXIST-1 and -2 sealed everolimus as a disease-modifying therapy across multiple TSC tumor types.

Related entities

Cite this page

ShowHide

Barton, O. (2026). Renal angiomyolipoma [Entity page]. In Oliver's mTOR Atlas [Data set]. https://mtor-atlas.org/disease/renal-angiomyolipoma/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_disease_renal_angiomyolipoma,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}: Renal angiomyolipoma},
  howpublished = {Entity page in a data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/disease/renal-angiomyolipoma/},
  doi          = {10.5281/zenodo.22059963}
}

Was this page useful?