Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis

Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN · 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 type	2 - Human Clinical Trial
Model system	Human (clinical trial)
Journal	The New England journal of medicine
Year	2008
Peer reviewed	Yes
Record last updated	2026-10-01
Source	DOI 10.1056/NEJMoa063564 · PMID 18184959 · Free full text (PMC3398441)

Extracted findings

These fields were extracted from the paper with AI assistance and have not been checked against the source. Read them as a pointer into the paper, not as curated claims; where they disagree with the curated summary above, the curated summary stands.

Intervention	Sirolimus (rapamycin)
Target	mTOR
Model	Human – open-label trial (TSC/LAM)
Effect	Sirolimus reduced angiomyolipoma volume during treatment; tumors regrew after stopping
Dose	Oral 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 size	Sirolimus reduced angiomyolipoma volume (~50%) during treatment; tumors regrew after the drug was stopped.
Limitations	Non-randomized open-label; benefit not durable off-drug; full text not retrievable, so figures from abstract only.

In the Atlas

Cite this paper

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Bissler, J. J., McCormack, F. X., Young, L. R., Elwing, J. M., Chuck, G., Leonard, J. M., Schmithorst, V. J., Laor, T., Brody, A. S., Bean, J., Salisbury, S., & Franz, D. N. (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 McCormack, F. X. and Young, L. R. and Elwing, J. M. and Chuck, G. and Leonard, J. M. and Schmithorst, V. J. and Laor, T. and Brody, A. S. and Bean, J. and Salisbury, S. and Franz, D. N.},
  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}
}

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