Table 1.
Patient baseline characteristics (n = 141)
| Characteristics | Median [range] or n (%) |
|---|---|
| Age at start mTORi (years) | 35.8 [17–76] |
| Male sex | 69 (48.9) |
| Mutation type | |
| TSC1 | 17 (12.1) |
| TSC2 | 63 (44.7) |
| VUS | 1 (0.7) |
| No mutation identified | 10 (7.1) |
| Unknown | 50 (35.5) |
| rAML | 127 (90.1) |
| SEGA | 46 (32.6) |
| Epilepsy | 120 (85) |
| LAM | 37 (26.2) |
| Duration use mTORi (years) | 5.1 [0.1–13.8] |
| mTORi typea | |
| Sirolimus | 17 |
| Everolimus | 139 |
a15 patients used sirolimus before switching to everolimus. mTORi, mechanistic target of rapamycin inhibitor; VUS, variant of unknown significance; rAML, renal angiomyolipoma; LAM, lymphangioleiomyomatosis; SEGA, subependymal giant-cell astrocytoma