Page "Lymphangioleiomyomatosis" Paragraph 34

Sirolimus has recently been tested for the treatment of LAM.

The MILES Trial ( Multicenter International LAM Efficacy of Sirolimus Trial ) was a randomized, double-blind, and placebo-controlled.

The primary endpoint was the rate of change in lung function over one year on treatment.

A total of 89 patients were enrolled at 13 sites in the United States, Japan and Canada.

To be eligible, patients had to have definite LAM and abnormal lung function.

In general, patients who enrolled had moderately severe LAM with lung function of about 50 % of predicted.

Patients were treated with sirolimus or placebo for one year and followed off drug for one year.

The data demonstrated that sirolimus stabilized lung function, improved some measures of quality of life and functional performance, and reduced serum VEGF-D in patients with LAM.

Side effects were more frequent in the sirolimus group, but serious adverse events were balanced between the groups.

The benefit of sirolimus waned when the drug was stopped, and lung function decline resumed.

The conclusion of the study was that sirolimus can be considered for treatment of moderately severe LAM.

Additional studies are needed to determine the risks and benefits of treatment in patients with milder disease and those who take the drug for longer than 1 year.