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 |