Table 2 Anthropometric data, lung function, sweat chloride concentration, and body composition parameters at baseline and 24 months of LUM/IVA treatment of patients who completed the follow-up period.

From: Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data

Characteristic

At baseline

24 months of LUM/IVA

p-value

Overall

2–5 years old

6–11 years old

 ≥ 12 years old

Overall

2–5 years old

6–11 years old

 ≥ 12 years old

Weight z-score*

− 0.54 (− 1.11, 0.16)

− 0.25 (− 1.03, 0.65)

− 0.32 (− 0.76, 0.14)

− 1.06 (− 1.86, − 0.11)

− 0.19 (− 0.84, 0.19)

− 0.19 (− 0.47, 0.54)

− 0.07 (− 0.79, 0.21)

− 0.50 (− 1.50, − 0.01)

0.074

Height z-score*

− 0.31 (− 1.04, 0.62)

− 0.06 (− 0.69, 0.60)

0.20 (− 0.65, 0.82)

− 0.65 (− 1.38, 0.01)

− 0.13 (− 0.68, 0.65)

− 0.20 (− 0.67, 0.23)

0.08 (− 0.69, 1.00)

− 0.13 (− 0.55, 0.18)

0.042

BMI z-score*

− 0.81 (− 1.37, 0.49)

− 0.29 (− 1.44, 0.79)

− 0.45 (− 0.93, 0.69)

− 1.00 (− 1.65, − 0.71)

− 0.39 (− 0.88, − 0.04)

− 0.03 (− 0.51, 0.65)

− 0.40 (− 0.82, − 0.12)

− 0.61 (− 1.53, − 0.23)

0.288

ppFEV1 (%)*

102 (82, 114)

–

112 (104, 122)

83 (72, 98)

87 (74, 96)

–

96 (88, 103)

76 (68, 88)

 < 0.001

Sweat chloride (mmol/L) *

75 (69, 82)

69 (64, 78)

75 (70, 77)

79 (68, 90)

57 (43, 70)

49 (37, 63)

60 (48, 76)

57 (44, 68)

 < 0.001

Fecal elastase (µg/g)

        

0.085

 < 100

28 (97%)

9 (100%)

10 (100%)

9 (90%)

23 (79%)

6 (67%)

9 (90%)

8 (80%)

 

 100–200

0 (0%)

0 (0%)

0 (0%)

0 (0%)

2 (7%)

1 (11%)

0 (0%)

1 (10%)

 

 > 200

1 (3%)

0 (0%)

0 (0%)

1 (10%)

4 (14%)

2 (22%)

1 (10%)

1 (10%)

 

Body composition (%)*

 Total body water

63 (59, 66)

63 (62, 66)

62 (58, 65)

64 (61, 67)

62 (57, 65)

60 (55, 63)

59 (58, 62)

64 (62, 68)

0.051

 Body protein content

17 (16, 18)

17 (16, 17)

16 (16, 18)

17 (16, 18)

17 (15, 17)

16 (14, 17)

16 (15, 17)

17 (17, 19)

0.087

 Fat mass

11 (9, 17)

8 (3, 17)

15 (10, 17)

11 (9, 16)

15 (12, 22)

19 (15, 25)

18 (15, 21)

12 (8, 14)

0.012

 Skeletal muscle mass

43 (39, 47)

40 (38, 43)

43 (39, 45)

48 (42, 50)

42 (40, 49)

39 (34, 42)

41 (40, 45)

49 (46, 51)

0.714

  1. LUM/IVA lumacaftor/ivacaftor, BMI body mass index, ppFEV1 percentile predicted forced expiratory volume in the first second.
  2. *median, interquartile range.
  3. Significant values are in bold.
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