Oliver's mTOR Atlas Evidence Platform
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Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis

Krueger DA et al. · 2010 · New England Journal of Medicine · Atlas ID KRU2010

What this study shows

In patients whose TSC1/TSC2 mutations cause brain tumors, everolimus shrank tumor volume by 30%+ in three-quarters of patients. Open-label and uncontrolled (n=28); randomised confirmation came later (FRA2013).

Abstract

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Neurosurgical resection is the standard treatment for subependymal giant-cell astrocytomas in patients with the tuberous sclerosis complex. An alternative may be the use of everolimus, which inhibits the mammalian target of rapamycin, a protein regulated by gene products involved in the tuberous sclerosis complex. Patients 3 years of age or older with serial growth of subependymal giant-cell astrocytomas were eligible for this open-label study. The primary efficacy end point was the change in volume of subependymal giant-cell astrocytomas between baseline and 6 months.

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, open-label trial (n=28)
JournalNew England Journal of Medicine
Year2010
Peer reviewedYes
Record last updated2026-07-29
SourceDOI 10.1056/NEJMoa1001671 · PMID 21047224

Extracted findings

InterventionEverolimus
TargetmTOR
ModelHuman – open-label trial (n=28, TSC)
EffectEverolimus reduced subependymal giant-cell astrocytoma (SEGA) volume in tuberous sclerosis

In the Atlas

Related topics

TSC1/TSC2mTOREverolimus

More studies on this topic

Cite this paper

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Krueger, D. A., et al. (2010). Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. New England Journal of Medicine. https://doi.org/10.1056/NEJMoa1001671

@article{KRU2010,
  author       = {Krueger, D. A. and others},
  title        = {{Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis}},
  journal      = {New England Journal of Medicine},
  year         = {2010},
  doi          = {10.1056/NEJMoa1001671},
  note         = {PMID: 21047224},
}

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 KRU2010) [Data set]. https://mtor-atlas.org/study/KRU2010/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_KRU2010,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record KRU2010},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/KRU2010/},
  doi          = {10.5281/zenodo.22059963}
}