Table 2.
Efficacy results
| ITT population | (n=17) | Baseline | 6 months | 12 months | 24 months |
|---|---|---|---|---|---|
|
50% reduction from baseline1 |
n (%) [95%CI] |
- |
11 (64.7%) |
14 (82.4%) |
10 (58.82%) |
| |
|
|
[38.3% to 85.8%] |
[56.6% to 96.2%] |
[32.92% to 81.56%] |
|
30% reduction from baseline2 |
n (%) [95%CI] |
- |
15 (88.2%) |
17 (100.0%) |
14 (82.4%) |
| |
|
|
[63.6% to 98.5%] |
[83.8% to 100.0%] |
[56.6% to 96.2%] |
|
Adjusted baseline change (%) |
LSMean [95%CI] |
0 |
−55.18 [−65.85 to −44.51] |
−66.38 [−75.77 to −56.98] |
−62.08 [−72.70 to −51.46] |
| |
6 vs 12 months |
- |
Ref |
−11.20 [−5.61 to −16.78] p<0.001 |
- |
| |
12 vs 24 months |
- |
- |
Ref |
4.30 [−2.61 to 11.21] p=0.205 |
|
Absolute values (cm3) |
LSMean [95%CI] |
62.65 [24.05 to 101.25] |
28.62 [12.91 to 44.33] |
23.07 [8.29 to 37.84] |
21.68 [11.30 to 32.06] |
| |
0 vs 6 months |
Ref |
−34.03 [−9.90 to −58.16] p=0.008 |
- |
- |
| |
0 vs 12 months |
Ref |
- |
−39.58 [−14.74 to −64.42] p=0.004 |
- |
| 0 vs 24 months | Ref | - | - | −40.97 [−11.15 to −70.79] p=0.010 |
Response of angiomyolipoma to rapamycin therapy: tumour volume and baseline reduction.
Figures for continuous variables are least square means (LSMean) and [95%CI] from the mixed models for repeated measurements (MMRM) analysis, for binary variables frequencies, (%) and [95%]. Values in the MMRM analysis are adjusted by the baseline volume, when specified.
1: Main predefined outcome in the protocol. 2: Reduction cut-off point according to the RECIST criteria.