Summary of rapamycin (sirolimus) trials for LAM
| Year | Type of study | Sample size | Intervention | Drug levels | Duration | Effects |
| 20083 | Open label | 11 (LAM) 20 (AML) | Sirolimus | 10–15 ng/mL except one case | 12 m treatment 12 m observation | ↑ FEV1, FVC ↓ AML size |
| 20114 7 | Randomised, double blind | 89 | Sirolimus versus placebo | 5–15 ng/mL | 12 m treatment 12 m observation | ↑ FEV1, FVC, QoL, functional performance ↓ VEGF-D, FRC, air trapping ↔ 6MWD, DLCO |
| 20119 | Observation | 19 | Sirolimus | 5–15 ng/mL | 2.6±1.2 years | ↑ FEV1, FVC, DLCO ↓ Chylothorax volume |
| 201113 | Observation | 10 | Sirolimus | 5–10 ng/mL | 12.1±2.8 m | ↑ FEV1, FVC, 6MWD, DLCO ↔ TLC, PaO2 |
| 201118 | Open label | 16 | Sirolimus | 3–10 ng/mL | 24 m | ↓ AML size ↔ FEV1, FVC, DLCO |
| 201312 | Retrospective | 15 | Sirolimus | 2.16 (0.8–4.3) ng/mL | 17.5±5.9 m without chylothorax 12.0±5.5 m with chylothorax | ↑ FEV1, FVC ↓ VEGF-D, chylothorax |
| 201419 | Observation | 38 | Sirolimus | 5–15 ng/mL | 3.4±2.4 years; 5 years in 12 patients | ↑ FEV1, DLCO ↔ Cysts, 6MWD |
| 201520 | Retrospective | 78 | Sirolimus, simvastatin or combined | Mean 2.7–2.8 years | ↑ FEV1, DLCO ↔ No effects from adding simvastatin | |
| 201611 | Open label | 63 | Sirolimus | 5–15 ng/mL | 24 m | ↑ FEV1, FVC in a chylothorax subgroup ↔ QoL, FEV1, FVC |
| 201610 | Observation | 25 | Sirolimus | 5–15 ng/mL | 4.5±1.6 years | ↓ Chylothorax, VEGF-D, AML size ↔ FEV1, DLCO |
6MWD, 6 min walk distance; AML, angiomyolipoma; DLCO, diffusing capacity of the lung for carbon monoxide; FEV1, forced expiratory volume in 1 s; FRC, functional residual capacity; FVC, forced vital capacity; LAM, lymphangioleiomyomatosis; m, months; QoL, quality of life; RV, residual volume; TLC, total lung capacity; TSC, tuberous sclerosis complex; VEGF-D, vascular endothelial growth factor-D; ↑, improved or increased in size or value; ↓, worsened or decreased in size or value; ↔, similar or unchanged.