Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial
This is a study to determine if early, long-term low dose sirolimus is effective for preventing progression to more advanced stages.
• Female, age 18 or over
• Signed and dated informed consent
• Diagnosis of LAM as determined by compatible lung CT and one of the following
‣ biopsy (lung, abdominal mass, lymph node or kidney) or cytology from thoracic or abdominal sources revealing LAM, or
⁃ tuberous sclerosis, angiomyolipomata (diagnosed by CT, MRI by the site radiologist or biopsy) or chylous pleural effusion (verified by tap), or
⁃ VEGF-D level ≥ 800 pg/ml.
• Post-bronchodilator forced expiratory volume in one second of \> 70%
• Presence of markers of non-trivial burden of LAM or likely progression based on one of the following:
‣ pretrial FEV 1 rate of decline of \>60cc/yr, comparing enrollment FEV1 to any prior measurement in the past 3 years, or
⁃ baseline supplemental oxygen requirement with exercise, or
⁃ pre-menopausal and one of the following (if post-menopausal, must have a VEGF-D level ≥ 600 pg/ml and one of the following) baseline diffusing capacity for carbon monoxide ≤80% predicted,
∙ a) baseline residual volume ≥120% predicted, b) baseline desaturation by 4% or more on six minute walk testing on room air c) more than 20 cysts on the carinal cut of the CT