Oliver's mTOR Atlas Evidence Platform
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Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis

Bissler JJ, Franz DN et al. · 2008 · The New England journal of medicine · Atlas ID BIS2008

What this study shows

Sirolimus shrinks renal angiomyolipomas in tuberous sclerosis / lymphangioleiomyomatosis.

Abstract

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Angiomyolipomas in patients with the tuberous sclerosis complex or sporadic lymphangioleiomyomatosis are associated with mutations in tuberous sclerosis genes resulting in constitutive activation of the mammalian target of rapamycin (mTOR). The drug sirolimus suppresses mTOR signaling. We conducted a 24-month, nonrandomized, open-label trial to determine whether sirolimus reduces the angiomyolipoma volume in patients with the tuberous sclerosis complex or sporadic lymphangioleiomyomatosis. Sirolimus was administered for the first 12 months only.

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 systemHuman (clinical trial)
JournalThe New England journal of medicine
Year2008
Peer reviewedYes
Record last updated2026-08-22
SourceDOI 10.1056/NEJMoa063564 · PMID 18184959 · Free full text (PMC3398441)

Extracted findings

InterventionSirolimus (rapamycin)
TargetmTOR
ModelHuman – open-label trial (TSC/LAM)
EffectSirolimus reduced angiomyolipoma volume during treatment; tumors regrew after stopping
DoseOral sirolimus, 24-month non-randomized open-label trial (dose titrated to therapeutic trough); full text not retrievable via PMC.
Sample size~25 patients with TSC or sporadic LAM (full text unavailable; abstract-level).
Effect sizeSirolimus reduced angiomyolipoma volume (~50%) during treatment; tumors regrew after the drug was stopped.
LimitationsNon-randomized open-label; benefit not durable off-drug; full text not retrievable, so figures from abstract only.

In the Atlas

Related topics

mTOR

Cite this paper

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Bissler, J. J., et al. (2008). Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. The New England journal of medicine. https://doi.org/10.1056/NEJMoa063564

@article{BIS2008,
  author       = {Bissler, J. J. and Franz, D. N. and others},
  title        = {{Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis}},
  journal      = {The New England journal of medicine},
  year         = {2008},
  doi          = {10.1056/NEJMoa063564},
  note         = {PMID: 18184959},
}

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 BIS2008) [Data set]. https://mtor-atlas.org/study/BIS2008/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_BIS2008,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record BIS2008},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/BIS2008/},
  doi          = {10.5281/zenodo.22059963}
}