Abstract | BACKGROUND: METHODS: RESULTS: Of the 25 patients enrolled, 20 completed the 12-month evaluation, and 18 completed the 24-month evaluation. The mean (+/-SD) angiomyolipoma volume at 12 months was 53.2+/-26.6% of the baseline value (P<0.001) and at 24 months was 85.9+/-28.5% of the baseline value (P=0.005). At 24 months, five patients had a persistent reduction in the angiomyolipoma volume of 30% or more. During the period of sirolimus therapy, among patients with lymphangioleiomyomatosis, the mean forced expiratory volume in 1 second (FEV1) increased by 118+/-330 ml (P=0.06), the forced vital capacity (FVC) increased by 390+/-570 ml (P<0.001), and the residual volume decreased by 439+/-493 ml (P=0.02), as compared with baseline values. One year after sirolimus was discontinued, the FEV1 was 62+/-411 ml above the baseline value, the FVC was 346+/-712 ml above the baseline value, and the residual volume was 333+/-570 ml below the baseline value; cerebral lesions were unchanged. Five patients had six serious adverse events while receiving sirolimus, including diarrhea, pyelonephritis, stomatitis, and respiratory infections. CONCLUSIONS:
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Authors | John J Bissler, Francis X McCormack, Lisa R Young, Jean M Elwing, Gail Chuck, Jennifer M Leonard, Vincent J Schmithorst, Tal Laor, Alan S Brody, Judy Bean, Shelia Salisbury, David N Franz |
Journal | The New England journal of medicine
(N Engl J Med)
Vol. 358
Issue 2
Pg. 140-51
(Jan 10 2008)
ISSN: 1533-4406 [Electronic] United States |
PMID | 18184959
(Publication Type: Clinical Trial, Phase I, Clinical Trial, Phase II, Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
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Copyright | 2008 Massachusetts Medical Society |
Chemical References |
- Immunosuppressive Agents
- Protein Kinase Inhibitors
- Protein Kinases
- MTOR protein, human
- TOR Serine-Threonine Kinases
- Sirolimus
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Topics |
- Adult
- Angiomyolipoma
(drug therapy, etiology, pathology)
- Brain
(pathology)
- Female
- Humans
- Immunosuppressive Agents
(adverse effects, therapeutic use)
- Kidney Diseases
(pathology)
- Liver Diseases
(pathology)
- Lung
(diagnostic imaging, physiopathology)
- Lung Neoplasms
(complications, pathology, physiopathology)
- Lymphangioleiomyomatosis
(complications, pathology, physiopathology)
- Magnetic Resonance Imaging
- Male
- Middle Aged
- Protein Kinase Inhibitors
(therapeutic use)
- Protein Kinases
(metabolism)
- Radiography
- Respiratory Function Tests
- Sirolimus
(adverse effects, therapeutic use)
- TOR Serine-Threonine Kinases
- Tuberous Sclerosis
(complications, genetics)
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