Table 3 Mean values of bone parameters at baseline and after 3, 6, 9, and 12 months of Lumacaftor/Ivacaftor treatment

Vitamin D (SD)

40.9 (± 24.5)

36.6 (± 19.2)

37.6 (± 25.9)

45.8 (± 18.5)

51.9 (± 24.3)

 N

8

6

4

4

8

 Change from baseline

-2.2

-12.6

3.6

9.2

 P-value

1.00

0.750

0.750

0.382

Phosphorus (SD)

3.7 (± 0.6)

3.8 (± 0.7)

3.6 (± 0.8)

3.5 (± 0.5)

3.3 (± 0.5)

 N

11

9

9

11

11

 Change from baseline

0.1

-0.1

-0.2

-4.9

 P-value

0.638

0.891

0.270

0.022

Calcium (SD)

9.5 (± 0.5)

9.6 (± 0.4)

9.9 (± 0.4)

9.8 (± 0.5)

9.7 (± 0.3)

 N

12

12

11

12

12

 Change from baseline

0.1

0.3

0.3

0.2

 P-value

0.570

0.074

0.106

0.142

PTH (SD)

40.9 (± 14.4)

38.4(± 10.1)

47.1 (± 14.6)

59.2(± 81.1)

39.7 (± 11.6)

 N

8

8

6

6

5

 Change from baseline

-2.8

1.2

-2.4

5

 P-value

0.546

1.00

0.843

0.250

Alkaline-phosphatase (SD)

122.8 (± 71)

89.4(± 49.2)

111.3(± 104)

108 (± 99.6)

106.8 (± 78.8)

 N

12

10

11

12

12

 Change from baseline

-37.5

-13.8

-14.8

-15.9

 P-value

0.002

0.051

0.075

0.176

 Relative change (%)

-28.1

-18.9

-18.8

-15.6

Urine Ca/Cr (SD)

0.1 (± 0.0)

0.1 (± 0.1)

0.2 (± 0.1)

0.2 (± 0.1)

0.1 (± 0.1)

 N

9

8

6

4

7

 Change from baseline

0

0.1

0.1

0

 P-value

0.664

0.062

0.125

0.593

DEXA: Spinal Total T-score

Mean (SD)

-2.1(± 0.5)

-1.6(± 0.4)

 N

4

4

 Change from baseline

0.5

 P-value

0.110