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Table 1 Endpoint behavioural outcomes

From: Randomised controlled trial of simvastatin treatment for autism in young children with neurofibromatosis type 1 (SANTA)

Week 12 outcomes

Summary statistics

Mean difference

 

Sample

Placebo

Simvastatin

Adjusted mean difference (95% CI)

Bootstrap SE

Effect size (95% CI)

Number analysed

ABC

N = 28

N = 15

N = 13

    

 Irritability

19.14 (11.62)

16.40 (10.82)

22.31 (12.14)

1.66 (− 4.61, 7.93)

3.20

0.14 (− 0.40, 0.68)

28

 Lethargy*

12.63 (10.01)

10.53 (9.61)

15.25 (10.30)*

3.60 (− 4.09, 11.28)

3.92

0.29 (− 0.41, 1.13)

26

 Stereotypy

5.71 (5.08)

3.93 (3.63)

7.77 (5.83)

1.61 (− 0.98, 4.20)

1.32

0.32 (− 0.19, 0.83)

28

 Hyperactivity

23.61 (13.68)

19.13 (13.17)

28.77(12.83)

3.87 (− 3.28, 11.01)

3.65

0.28 (− 0.24, 0.80)

28

 Inappropriate speech

5.89 (3.11)

4.80 (2.54)

7.15 (3.31)

1.77 (− 0.10, 3.63)

0.95

0.57 (− 0.03, 1.17)

28

25% reduction irritability subscale

N = 11

N = 5

N = 6

    

Conners

N = 28

N = 15

N = 13

    

 Inattention

77.25 (12.57)

74.53 (14.16)

80.38 (10.08)

5.33 (− 0.96, 11.61)

3.21

0.42 (− 0.08, 0.92)

27

 Hyperactivity

73.54 (15.92)

69.40 (17.12)

78.31 (13.51)

−0.98 (− 8.09, 6.13)

3.63

−0.06 (− 0.51, 0.39)

27

 Learning problems

69.25 (14.62)

65.40 (12.91)

73.69 (15.71)

1.59 (− 2.13, 5.30)

1.89

0.11 (− 0.15, 0.36)

27

 Executive function

71.82 (14.59)

68.20 (16.24)

76.00 (11.66)

4.04 (− 2.51, 10.59)

3.34

0.28 (− 0.17, 0.73)

27

 Aggression

70.43 (18.98)

68.40 (20.86)

72.77 (17.09)

− 0.05 (− 11.05,10.96)

5.61

− 0.00 (− 0.58, 0.58)

27

 Peer relations

83.89 (11.53)

82.00 (13.51)

86.08 (8.73)

1.38 (− 4.45, 7.21)

2.97

0.12 (− 0.39, 0.63)

27

Parent-defined target symptoms (PDTS)

N = 26

N = 14

N = 12

    

Mean (SD)

3.52 (1.77)

3.75 (1.86)

3.25 (1.68)

    

Responders (PDTS score < 3)

7

2

5

    

CGI*

N = 26

N = 14

N = 12

    

 Global improvement Mean (SD)

3.31 (0.84)

3.57 (0.85)

3.00 (0.74)

    

Treatment responder+

N = 3

N = 0

N = 3

    
  1. *1 additional observation missing. +Treatment responder defined as > 25% reduction in ABC irritability subscale and a score of improved or much improved on CGI
  2. Higher scores on ABC, Conners and CGI are indicative of higher levels of impairment