Table 2 Patient demographics and baseline characteristics

N

26

29

55

Female, number (%)

13 (50.0%)

15 (51.7%)

28 (50.9%)

Male, number (%)

13 (50.0%)

14 (48.3%)

27 (49.1%)

Age at enrolment, years

 N

26

29

55

 Mean (SD)

14.4 (11.43)

15.3 (15.30)

14.9 (13.50)

 Median

9

8

9

 Min, max

4, 41

2, 58

2, 58

Weight (kg)

 N

26

25

51

 Mean (SD)

28.0 (13.16)

25.04 (15.72)

26.6 (14.58)

 Median

23.8

16.5

21.7

 Min, max

14.2, 65.0

10.3, 62.6

10.3, 65.0

Treatment duration, years*

 N

26

29

55

 Mean (SD)

7.46 (1.62)

2.36 (0.88)

4.78 (2.86)

 Median

6.96

2.98

3.00

 Min, max

4.08, 9.54

0.99, 3.04

0.99, 9.54

6MWT, m

 N

23

24

47

 Mean (SD)

220.1 (91.47)

193.5 (105.80)

206.7 (98.86)

 Median

228

184.5

215

 Min, max

60, 433

20, 420

20, 433

FVC, L

 N

25

18

43

 Mean (SD)

0.9 (0.66)

1.2 (0.97)

1.0 (0.80)

 Median

0.6

0.8

0.7

 Min, max

0.3, 2.9

0.3, 3.9

0.3, 3.9

FEV₁, L

 N

25

18

43

 Mean (SD)

0.8 (0.55)

0.9 (0.69)

0.8 (0.61)

 Median

0.6

0.7

0.6

 Min, max

0.2, 2.5

0.3, 2.4

0.2, 2.5

LVEF, %

 N

21

22

43

 Mean (SD)

66 (6)

66 (10)

65 (8.2)

 Median

65

69

65

 Min, max

55, 80

36, 82

36, 82

uKS, µg/mg creatinine

 N

26

25

51

 Mean (SD)

28.0 (16.01)

37.5 (23.3)

32.7 (20.27)

 Median

32.4

44.3

35.1

 Min, max

3.5, 50.3

2.6, 74.7

2.6, 74.7