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Table 2 Shows the baseline characteristics of the included studies

From: Safety and efficacy of trofinetide in Rett syndrome: a systematic review and meta-analysis of randomized controlled trials

Study ID

Glaze et al., 2017

Glaze et al., 2019

Neul et al., 2023

 

Trofinetide

Placebo

Trofinetide

Placebo

Trofinetide

Placebo

 

35 mg/kg

70 mg/kg

 

50 mg/kg

100 mg/kg

200 mg/kg

 

200 mg/kg

 

Number of patients (n)

13

17

Doses: (35 mg/kg) = 5, (70 mg/kg) = 11

15

16

27

24

93

94

Gender [Female n (%)]

30 (100%)

16 (100%)

58 (100%)

24 (100%)

93 (100%)

94 (100%)

Age, year [Mean (SD)]

22.62 (5.582)

24.52 (5.853)

Doses: (35 mg/kg) = 32.09 (9.324), (70 mg/kg) = 27.09 (8.357)

10.06 (3.18)

10.81 (3.10)

9.23 (3.88)

9.38 (3.26)

11.0 (4.69)

10.9 (4.57)

BMI, kg/cm2 [Mean (SD)]

25.06 (7.930)

20.48 (6.765)

Doses: (35 mg/kg) = 24.66 (8.04), (70 mg/kg) = 19.24 (3.598)

16.50 (3.61)

17.70 (5.06)

16.31 (3.57)

16.00 (2.85)

  

Ethnicity, n (%)

         

Hispanic

0

2 (12%)

Doses: (35 mg/kg) = 1 (20%), (70 mg/kg) = 0

1 (7%)

1 (6%)

6 (22%)

0

  

Not Hispanic

13 (100%)

15 (88%)

Doses: (35 mg/kg) = 4 (80%), (70 mg/kg) = 11 (100%)

14 (93%)

14 (88%)

21 (78%)

24 (100%)

  

Race, n (%)

         

Asian

0

1 (6%)

Doses: (35 mg/kg) = 0, (70 mg/kg) = 0

0

0

2 (7%)

1 (4%)

5 (5.4%)

1 (1.1%)

Black or African-American

3 (23%)

1 (6%)

Doses: (35 mg/kg) = 0, (70 mg/kg) = 0

0

1 (6%)

0

0

1 (1.1%)

1 (1.1%)

White

10 (77%)

15 (88%)

Doses: (35 mg/kg) = 5 (100%), (70 mg/kg) = 11 (100%)

15 (100%)

15 (94%)

25 (93%)

22 (92%)

82 (88.2%)

90 (95.7%)

Other

0

  

0

0

0

1 (4%)

4 (4.3%)

2 (2.1%)