Table 1. Patient baseline characteristics.
| Characteristic | Everolimus N = 112 |
|---|---|
| Age, median (range), years | 32.2 (18.1–61.6) |
| Age category, years, n (%) | |
| <30 | 49 (43.8) |
| ≥30 | 63 (56.3) |
| Sex, n (%) | |
| Male | 39 (34.8) |
| Female | 73 (65.2) |
| Race, n (%) | |
| White | 99 (88.4) |
| Asian | 11 (9.8) |
| Othera | 2 (1.8) |
| Diagnosis of TSC,b n (%) | 107 (95.5) |
| Diagnosis of sporadic LAM, n (%) | 5 (4.5) |
| Diagnosis of LAM, n (%) | 24 (21.4) |
| Presence of ≥1 SEGA lesion, n (%) | 50 (44.6) |
| Presence of ≥1 skin lesion, n (%) | 107 (95.5) |
| Facial angiofibromas or forehead plaque | 105 (93.8) |
| Ungual or periungual fibroma | 68 (60.7) |
| Hypomelanotic macules (three or more) | 69 (61.6) |
| Shagreen patch | 54 (48.2) |
| 'Confetti' skin lesions | 30 (26.8) |
| Prior renal angiomyolipoma—related surgery, n (%) | 42 (37.5) |
| Prior nephrectomy, n (%) | 21 (18.8) |
| Bilateral renal angiomyolipoma lesions, n (%) | 88 (78.6) |
| Longest diameter of largest renal angiomyolipoma lesion, n (%) | |
| ≥8 cm | 33 (29.5) |
| ≥4 cm and <8 cm | 64 (57.1) |
| ≥3 cm and <4 cm | 7 (6.3) |
| <3 cm | 6 (5.4) |
| Unknown | 0 |
| Not applicable | 2 (1.8) |
| Target renal angiomyolipoma lesions ≥1 cm, n (%) | |
| 0 | 2 (1.8) |
| 1–5 | 43 (38.4) |
| 6–10 | 67 (59.8) |
| Sum of volumes of target renal angiomyolipoma lesions | |
| Number of patients with ≥1 target renal angiomyolipoma | 110 |
| Median (range), cm3 | 92.1 (2.8–1611.5) |
Abbreviations: LAM = lymphangioleiomyomatosis; SEGA = subependymal giant cell astrocytoma; TSC = tuberous sclerosis complex.
aDesignates patients who were of mixed race.
bAll patients with diagnosed TSC had ≥2 major features of TSC. In patients with both LAM and renal angiomyolipoma, another feature must have been identified to assign TSC diagnosis.