Abstract | PURPOSE: EXPERIMENTAL DESIGN: In this multicenter phase 2 nonrandomized open label trial, 16 patients with tuberous sclerosis or sporadic LAM and renal angiomyolipoma(s) were treated with oral sirolimus for up to 2 years. Steady-state blood levels were 3 to 10 ng/mL. The primary outcome was change in size of renal angiomyolipomas measured by MRI and assessed by Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Secondary outcomes included safety, neurocognitive function, and pulmonary function. RESULTS: The response rate, by RECIST criteria, was 50%. Summated angiomyolipoma diameters were reduced in all 16 patients and by 30% or more in eight (all from the per protocol group of 10). Forty-one of 48 angiomyolipomas were smaller at the last measurement than at baseline. Most shrinkage occurred during the first year of treatment. There was little change in pulmonary function. Recall memory improved in seven of eight patients with tuberous sclerosis. Adverse events were consistent with the known toxicities of sirolimus. CONCLUSIONS: This study showed sustained regression of renal angiomyolipomas in patients with tuberous sclerosis or sporadic LAM receiving 2 years of sirolimus treatment. Possible effects on pulmonary function and neurocognition require further investigation.
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Authors | D Mark Davies, Petrus J de Vries, Simon R Johnson, Deborah L McCartney, Jane A Cox, Andreas L Serra, Peter C Watson, Christopher J Howe, Tim Doyle, Kate Pointon, Justin J Cross, Anne E Tattersfield, J Chris Kingswood, Julian R Sampson |
Journal | Clinical cancer research : an official journal of the American Association for Cancer Research
(Clin Cancer Res)
Vol. 17
Issue 12
Pg. 4071-81
(Jun 15 2011)
ISSN: 1557-3265 [Electronic] United States |
PMID | 21525172
(Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
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Copyright | ©2011 AACR. |
Chemical References |
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Topics |
- Adolescent
- Adult
- Aged
- Angiomyolipoma
(complications, drug therapy, pathology)
- Female
- Humans
- Lung
(physiopathology)
- Lymphangioleiomyomatosis
(complications, pathology)
- Male
- Middle Aged
- Neuropsychological Tests
- Sirolimus
(adverse effects, therapeutic use)
- Treatment Outcome
- Tuberous Sclerosis
(complications, pathology)
- Young Adult
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