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New therapy found for rare lung disorder
Date:3/16/2011

ated with improvement in measures of functional performance and quality of life.

Sirolimus also reduced levels of serum vascular endothelial growth factor-D, or VEGF-D, a protein that is known to be elevated in LAM. VEGF-D promotes the growth of lymphatic vessels and can be involved in the spread of cancers.

"After discontinuation of sirolimus, lung function decline resumed and paralleled the placebo group," McCormack says. "Adverse events were more common with sirolimus, but the frequency of serious adverse events between the groups was not different."

McCormack says that these results suggest that sirolimus may be useful as therapy for moderately severe LAM-related lung disease.

"LAM is typically slowly progressive, and sirolimus therapy has risks, so treatment decisions should be individualized. Care must be taken in generalizing the results to those with milder or more severe lung disease due to LAM," he says. "Also, additional trials are needed to determine the optimal dose and duration of treatment with sirolimus. Given the toxicity profile of the drug during a one-year period, the long-term safety of this approach over an extended course must be carefully evaluated."

The trial involved efforts by the LAM Foundation that lobbied for the NIH's attention, organized and recruited patients and supported pivotal basic and clinical research that formed the scientific basis for the study.


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Contact: Katie Pence
katie.pence@uc.edu
513-558-4561
University of Cincinnati Academic Health Center
Source:Eurekalert

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