Oliver's mTOR Atlas Evidence Platform
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Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial

Bissler JJ, Kingswood JC, Radzikowska E, Zonnenberg BA, Frost M, Budde K · 2013 · Lancet · Atlas ID BIS2013

What this study shows

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.

Abstract

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Angiomyolipomas are slow-growing tumours associated with constitutive activation of mammalian target of rapamycin (mTOR), and are common in patients with tuberous sclerosis complex and sporadic lymphangioleiomyomatosis. The insidious growth of these tumours predisposes patients to serious complications including retroperitoneal haemorrhage and impaired renal function. Everolimus, a rapamycin derivative, inhibits the mTOR pathway by acting on the mTOR complex 1.

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 type2 - Human Clinical Trial
Model systemHumans, phase 3 RCT (n=118, TSC/LAM)
JournalLancet
Year2013
Peer reviewedYes
Record last updated2026-08-22
SourceDOI 10.1016/S0140-6736(12)61767-X · PMID 23312829

Extracted findings

InterventionEverolimus
TargetmTORC1
ModelHuman – phase 3 RCT (EXIST-2, n=118, TSC/LAM)
EffectEverolimus reduced angiomyolipoma size vs placebo in TSC/LAM

In the Atlas

Related topics

LymphangioleiomyomatosisEverolimusmTORC1Tuberous sclerosis complex

Answers that reference this study

More studies on this topic

Cite this paper

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Bissler, J. J., Kingswood, J. C., Radzikowska, E., Zonnenberg, B. A., Frost, M., & Budde, K. (2013). Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet. https://doi.org/10.1016/S0140-6736(12)61767-X

@article{BIS2013,
  author       = {Bissler, J. J. and Kingswood, J. C. and Radzikowska, E. and Zonnenberg, B. A. and Frost, M. and Budde, K.},
  title        = {{Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial}},
  journal      = {Lancet},
  year         = {2013},
  doi          = {10.1016/S0140-6736(12)61767-X},
  note         = {PMID: 23312829},
}

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 BIS2013) [Data set]. https://mtor-atlas.org/study/BIS2013/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_BIS2013,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record BIS2013},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/BIS2013/},
  doi          = {10.5281/zenodo.22059963}
}