Table 2.
Baseline values with subgroup comparison (SMA type 2 versus SMA type 3)
| SMA | nBL | nBL↑ | nBL↓ | Meana ± SDa | Rangea | p valueb | |
|---|---|---|---|---|---|---|---|
| CSF | |||||||
|
WBC (/µL) |
2 | 13c | 1 | n/a | 2.3 ± 4.3 | 0–16 | |
| 3 | 34c | 2 | n/a | 2.4 ± 2.4 | 0–13 | ||
| Total | 47c | 3 | n/a | 2.4 ± 3.0 | 0–16 | 0.15 | |
|
Glucose (mg/dL) |
2 | 13c | n/a | 0 | 62.5 ± 5.8 | 53–72 | |
| 3 | 34c | n/a | 0 | 65.7 ± 5.4 | 56–75 | ||
| Total | 47c | n/a | 0 | 64.8 ± 5.7 | 53–75 | 0.12 | |
|
Lactate (mmol/L) |
2 | 13c | 0 | n/a | 1.35 ± 0.19 | 1.0–1.7 | |
| 3 | 34c | 1 | n/a | 1.57 ± 0.27 | 1.1–2.5 | ||
| Total | 47c | 1 | n/a | 1.51 ± 0.27 | 1.0–2.5 | 0.01 | |
|
Protein (mg/dL) |
2 | 13c | 1 | 0 | 34.1 ± 8.6 | 19–46 | |
| 3 | 34c | 11 | 0 | 41.9 ± 12.4 | 26–72 | ||
| Total | 47c | 12 | 0 | 39.7 ± 11.9 | 19–72 | 0.07 | |
| Blood | |||||||
|
WBC (/nL) |
2 | 13 | 2 | 0 | 7.2 ± 2.2 | 3.9–11.7 | |
| 3 | 35 | 5 | 0 | 7.1 ± 1.8 | 4.6–11.9 | ||
| Total | 48 | 7 | 0 | 7.1 ± 1.9 | 3.9–11.9 | 0.94 | |
|
CRP (mg/dL) |
2 | 13 | 5 | n/a | – | < 0.5–1.5 | |
| 3 | 31 | 10 | n/a | – | < 0.5–2.3 | ||
| Total | 44 | 15 | n/a | – | < 0.5–2.3 | 0.68 | |
|
Platelets (/nL) |
2 | 13 | 5 | 2 | 315.2 ± 122.0 | 103–477 | |
| 3 | 35 | 0 | 0 | 265.9 ± 50.9 | 176–368 | ||
| Total | 48 | 5 | 2 | 279.3 ± 78.5 | 103–477 | 0.07 | |
| INR | 2 | 12 | 0 | n/a | 1.02 ± 0.06 | 0.92–1.12 | |
| 3 | 16 | 1 | n/a | 1.02 ± 0.08 | 0.93–1.19 | ||
| Total | 28 | 1 | n/a | 1.02 ± 0.07 | 0.92–1.19 | 0.96 | |
|
aPTT (s) |
2 | 12 | 0 | n/a | 28.8 ± 2.6 | 24.3–32.1 | |
| 3 | 16 | 1 | n/a | 27.5 ± 3.1 | 20.9–32.9 | ||
| Total | 28 | 1 | n/a | 28.1 ± 2.9 | 20.9–32.9 | 0.29 | |
|
Crea (mg/dL) |
2 | 13 | 0 | 13 | 0.31 ± 0.05 | 0.26–0.43 | |
| 3 | 33 | 0 | 24 | 0.49 ± 0.14 | 0.28–0.78 | ||
| Total | 46 | 0 | 37 | 0.44 ± 0.15 | 0.26–0.78 | 0.0001 | |
|
Urea (mg/dL) |
2 | 13 | 0 | 2 | 8.8 ± 4.1 | 4.0–18.0 | |
| 3 | 33 | 3 | 1 | 11.9 ± 4.3 | 5.0–22.0 | ||
| Total | 46 | 3 | 3 | 11.0 ± 4.5 | 4.0–22.0 | 0.02 | |
|
AST (U/L) |
2 | 13 | 0 | n/a | 21.0 ± 8.9 | 11–39 | |
| 3 | 33 | 1 | n/a | 28.5 ± 13.1 | 11–72 | ||
| Total | 46 | 1 | n/a | 26.4 ± 12.4 | 11–72 | 0.07 | |
|
ALT (U/L) |
2 | 13 | 2 | n/a | 31.5 ± 29.1 | 8–111 | |
| 3 | 33 | 14 | n/a | 47.7 ± 24.2 | 17–118 | ||
| Total | 46 | 16 | n/a | 43.2 ± 26.4 | 8–118 | 0.01 | |
|
GGT (U/L) |
2 | 13 | 2 | n/a | 36.1 ± 45.1 | 7–176 | |
| 3 | 33 | 7 | n/a | 43.7 ± 39.3 | 7–190 | ||
| Total | 46 | 9 | n/a | 41.5 ± 40.7 | 7–190 | 0.14 | |
CSF cerebrospinal fluid, SMA spinal muscular atrophy, WBC white blood cell count, CRP C-reactive protein, INR international normalized ratio, aPTT activated partial thromboplastin time, crea creatinine, urea urea nitrogen, AST aspartate aminotransferase, ALT alanine aminotransferase, GGT gamma-glutamyltransferase, nBL number of available baseline values, nBL↑ number of elevated baseline values, nBL↓ number of lowered baseline values, SD standard deviation, n/a not applicable (only medically reasonable or meaningful directions of deviation were considered)
aUnits as specified in the first column
bSMA type 2 versus SMA type 3
cThree CSF baseline samples were excluded from statistical analyses because of red blood cell contamination