Clinical data for cohorts studied
| UK discovery cohort | UK replication cohort | UK rapamycin-treated group | USA NHLBI cohort | Healthy controls | |||
| All | Stable | Progressive | |||||
| Subjects n | 50 | 26 | 24 | 27 | 24 | 152 | 22 |
| Age years | 50.6±10.9 | 50.9±11.8 | 50.3±10.0 | 49.4±13.9 | 46.4±9.7 | 45.4±9.0 | 35.0±11.7 |
| Disease duration years | 13.9±11.1 | 14.2±11.4 | 13.5±11.1 | 9.1±9.5 | 13.1±9.5 | 4.6±4.3 | NA |
| Angiomyolipoma | 72 | 77 | 67 | 55 | 54 | NT | NA |
| Lymphatic disease | 16 | 15 | 17 | 23 | 25 | NT | NA |
| TSC | 14 | 19 | 8 | 15 | 21 | NT | NA |
| Pneumothorax | 48 | 50 | 46 | 40 | 46 | NT | NA |
| Post-menopause | 34 | 42 | 25 | 30 | 25 | 48 | NA |
| FEV1 % pred | 68.9±20.6 | 76.4±18.9 | 60.8±19.5 | 77.4±23.4 | 46.7±14.8 | 74.1±27.5 | NA |
| DLCO % pred | 59.8±15.8 | 68.9±12.7 | 50.0±12.9 | 62.9±17.1 | 43.3±12.3 | 55.7±25.6 | NA |
| VEGF-D pg·mL−1 | 1327±1187 | 985±833 | 1698±1405 | 1275±1527 | 1082±1257 | NT | 397±125 |
Data are presented as mean±sd (at recruitment) or % (present at any time in disease course), unless otherwise stated. NHLBI: National Heart, Lung and Blood Institute; TSC: tuberous sclerosis complex; FEV1: forced expiratory volume in 1 s; DLCO: diffusing capacity of the lung for carbon monoxide; VEGF: vascular endothelial growth factor; NA: not applicable; NT: not available for testing. Disease duration in the UK lymphangioleiomyomatosis cohort was from first symptom to enrolment, while in the NHLBI cohort disease duration was from diagnosis to enrolment. In the NHLBI cohort menopause was assumed if ≥50 years of age.