| TSC1 | TSC2 | P value |
---|---|---|---|
(n = 4) | (n = 68) | TSC1 versus TSC2 | |
AML volume at baseline (mean ± SD, cm3) | 70 ± 76 | 235 ± 538 | 0.54 |
Age range (years) | 29–45 | 16–59 | 0.079 |
 Median age (years) | 36 | 34 |  |
Sex | Â | Â | 0.63 |
 Male | 2 | 25 |  |
 Female | 2 | 43 |  |
Familial/de novo | Â | Â | 0.17 |
 Familial | 2 | 12 |  |
 De novo | 2 | 56 |  |
Everolimus (3Â months) | Â | Â | Â |
 No. of response (%) | 3 (75) | 52 (76) | 0.99 |
 % reduction from baseline value (mean ± SD, %) | 39 ± 14 | 38 ± 17 | 0.89 |
Everolimus (6Â months) | Â | Â | Â |
 No. of response (%) | 4 (100) | 56 (85) | 0.99 |
 % reduction from baseline value (mean ± SD, %) | 41 ± 15 | 43 ± 18 | 0.87 |