Study on Sirolimus for LAM Patients
Lymphangioleiomyomatosis (*LAM*) is a rare disease that affects mostly women, causing lung problems. This study, called MIDAS, will look at how safe and effective the drug sirolimus is for treating LAM over a long time. Sirolimus is part of a group of drugs called *mTOR inhibitors*, which help slow down lung damage. The study will include both men and women who take or might take sirolimus or everolimus, another mTOR inhibitor. Researchers want to see if taking sirolimus for a long time can help prevent LAM from getting worse. The study will track 600 patients, collecting lung function tests and checking for any side effects. Patients will be observed for at least two years. This will help doctors understand if sirolimus should be taken for life to manage LAM.
- Study is observational and lasts at least 2 years.
- No specific treatment given, but usual care is followed.
- Participants must visit clinic at least once a year.
Eligible participants are 18 or older, with a LAM diagnosis, and agree to take part in the study.