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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