Table 2.
Comparison of mean measures across all time points and pairwise comparisons for changes from time point 1 to follow-up time points
| 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 |
CI confidence interval, CSF cerebrospinal fluid, MPS mucopolysaccharidosis.