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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.