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Table 2 Efficacy results

From: Assessing the effectiveness of rapamycin on angiomyolipoma in tuberous sclerosis: a two years trial

ITT population (n=17) Baseline 6 months 12 months 24 months
50% reduction from baseline 1 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 baseline 2 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
  1. Response of angiomyolipoma to rapamycin therapy: tumour volume and baseline reduction.
  2. 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.
  3. 1: Main predefined outcome in the protocol. 2: Reduction cut-off point according to the RECIST criteria.