Table 3.
Shifts to abnormal laboratory values
| Infantile-onset SMA (ENDEAR) | Later-onset SMA (CHERISH) | |||
|---|---|---|---|---|
| Parameter, n/N (%) | Nusinersen-treated N = 80 |
Control-treated N = 41 |
Nusinersen-treated N = 84 |
Control-treated N = 42 |
| High blood urea nitrogen | 1/73 (1) | 0/34 | 2/84 (2) | 1/42 (2) |
| High creatinine | 0/73 | 0/34 | 1/84 (1) | 0/42 |
| High cystatin C | 1/73 (1) | 1/33 (3) | 1/83 (1) | 0/42 |
| Urinary protein (≥ 1 positivea result)b | 7/66 (11) | 6/32 (19) | 14/83 (17) | 6/41 (15) |
| Urinary protein (≥ 2 positivea results)c | 1/55 (2) | 0/23 | 1/83 (1) | 1/41 (2) |
| Low platelets | 9/70 (13) | 0/33 | 15/76 (20) | 10/39 (26) |
| High total bilirubin (direct) | 0/68 | 0/30 | 0/84 | 0/42 |
| High total bilirubin (indirect) | 0/68 | 0/28 | 0/84 | 1/42 (2) |
| High alkaline phosphatase | 3/72 (4) | 0/32 | 0/84 | 0/42 |
| High ALT | 10/69 (14) | 4/31 (13) | 3/82 (4) | 4/42 (10) |
| High AST | 5/70 (7) | 1/31 (3) | 2/83 (2) | 2/42 (5) |
ALT alanine aminotransferase, AST aspartate aminotransferase
aDefined as ≥ 1 + on urine dipstick
bThe number at risk for shift to positive is the number of participants whose baseline values were not positive and who had at least one post-baseline lab assessment
cThe number at risk for shift to positive is the number of participants whose baseline values were not positive and who had at least two post-baseline lab assessments