Table 2 Comparison of mean measures across all time points and pairwise comparisons for changes from time point 1 to follow-up time points

From: Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes

Time point

Mean (SD)

Δ From time point 1 (95% CI)

p value

Opening pressure (cm H2O; ref range ≤ 20 cm H2O) (ref. 32)

  1 (N = 23)

24.2 (7.4)

-

-

  2 (N = 23)

19.7 (5.3)

−4.5 (−8.6, −0.4)

0.0499

  3 (N = 20)

18.5 (3.7)

−5.7 (−8.6, −2.8)

0.0004

  4 (N = 19)

21.0 (3.7)

−3.2 (−6.8, 0.5)

0.0861

  Any time point

-

-

0.0003

CSF protein (mg/dL; non-MPS ref 15–60 mg/dL (ref. 27)

  1 (N = 23)

31.0 (13.9)

-

-

  2 (N = 24)

30.8 (10.7)

−0.3 (−5.4, 4.7)

0.8951

  3 (N = 20)

33.9 (12.6)

2.2 (−3.7, 8.1)

0.8951

  4 (N = 21)

33.0 (15.9)

1.1 (−6.7, 8.9)

0.8951

  Any time point

-

-

0.7760

CSF glucose (mg/dL; non-MPS ref 40–70 mg/dL (ref. 27)

  1 (N = 23)

48.8 (4.8)

-

-

  2 (N = 24)

46.2 (5.3)

−2.6 (−4.3, −1.0)

0.0058

  3 (N = 21)

48.7 (7.2)

−0.1 (−2.4, 2.3)

0.9536

  4 (N = 21)

51.0 (8.8)

2.3 (−1.6, 6.2)

0.3834

  Any time point

-

-

0.0150

CSF antidrug antibodies

  1 (N = 23)

0.012 (0.041)

-

-

  2 (N = 23)

0.082 (0.085)

0.069 (0.034, 0.104)

0.0003

  3 (N = 20)

0.003 (0.003)

−0.009 (−0.024, 0.006)

0.2534

  4 (N = 19)

0.002 (0.002)

−0.010 (−0.026, 0.005)

0.2534

  Any time point

-

-

<0.0001

I0S6 (ng/mL; non-MPS ref < 30 ng/mL (ref. 27)

  1 (N = 20)

240.4 (100.4)

-

-

  2 (N = 20)

157.9 (76.6)

−82.5 (−112.2, −52.8)

<0.0001

  3 (N = 20)

127.2 (55.5)

−113.2 (−145.9, −80.5)

<0.0001

  4 (N = 20)

110.1 (45.9)

−130.3 (−166.0, −94.6)

<0.0001

  Any time point

-

-

<0.0001

I0S0 (ng/mL; non-MPS ref < 15 ng/mL (ref. 27)

  1 (N = 20)

58.2 (32.0)

-

-

  2 (N = 20)

29.9 (15.4)

−28.3 (−42.0, −14.6)

<0.0001

  3 (N = 20)

16.8 (12.8)

−41.5 (−56.9, −26.0)

<0.0001

  4 (N = 20)

11.2 (10.6)

−47.1 (−61.2, −32.9)

<0.0001

  Any time point

-

-

<0.0001

Heparan sulfate (ng/mL; non-MPS ref < 120 ng/mL (ref. 27)

  1 (N = 20)

283.7 (99.5)

-

-

  2 (N = 20)

262.9 (80.9)

−20.8 (−60.1, 18.6)

0.3008

  3 (N = 20)

212.9 (83.8)

−70.8 (−118.0, −23.5)

0.0050

  4 (N = 20)

195.1 (60.2)

−88.6 (−131.2, −46.0)

0.0001

  Any time point

-

-

0.0001

Heparin cofactor II–thrombin (ng/mL; non-MPS ref < 0.25 ng/mL (ref. 27)

  1 (N = 21)

7.1 (3.1)

-

-

  2 (N = 23)

4.8 (3.5)

−2.3 (−3.6, −1.0)

0.0006

  3 (N = 21)

2.8 (1.9)

−4.4 (−5.7, −3.1)

<0.0001

  4 (N = 21)

3.3 (3.1)

−3.8 (−5.7, −2.0)

<0.0001

  Any time point

-

-

<0.0001

MCP-1 (pg/mL; no reference range (ref. 27)

  1 (N = 10)

976.7 (401.1)

-

-

  2 (N = 10)

891.4 (526.7)

−85.3 (−257.5, 86.9)

0.3317

  3 (N = 10)

760.9 (197.2)

−215.8 (−477.2, 45.6)

0.1586

  4 (N = 10)

595.7 (169.5)

−381.0 (−641.5, −120.5)

0.0124

  Any time point

-

-

0.0006

MIP-1b (pg/mL; no reference range27)

  1 (N = 10)

15.0 (7.0)

-

-

  2 (N = 10)

10.8 (3.6)

−4.2 (−7.5, −0.9)

0.0205

  3 (N = 10)

14.1 (7.2)

−0.9 (−6.3, 4.5)

0.7370

  4 (N = 10)

9.0 (4.8)

−6.0 (−10.2, −1.8)

0.0162

  Any time point

-

-

0.0070

SDF1 (pg/mL; no reference range27)

  1 (N = 10)

385.1 (94.7)

-

-

  2 (N = 10)

351.7 (123.0)

−33.3 (−144.9, 78.2)

0.8374

  3 (N = 10)

334.9 (168.7)

−50.1 (−179.4, 79.2)

0.8374

  4 (N = 10)

387.7 (303.1)

2.6 (−192.5, 197.8)

0.9788

  Any time point

-

-

0.5378

IL-1Ra (pg/mL; no reference range27)

  1 (N = 10)

18.7 (6.6)

-

-

  2 (N = 10)

25.0 (32.7)

6.3 (−12.8, 25.3)

0.5203

  3 (N = 10)

34.1 (33.5)

15.3 (−3.1, 33.8)

0.2497

  4 (N = 10)

32.0 (31.8)

13.2 (−5.5, 31.9)

0.2497

  Any time point

-

-

0.2759

  1. CI confidence interval, CSF cerebrospinal fluid, MPS mucopolysaccharidosis.