TABLE 1.
Baseline patient characteristics stratified by SMA type and for the overall sample
| Characteristic | Type 1 SMA (n = 28) | Type 2/3 SMA (n = 63) | Overall sample (N = 91) |
|---|---|---|---|
| Age at visit (y) | |||
| Median (IQR) | 1.1 (0.5–1.8) | 6.8 (4.0–11.4) | 5.5 (1.7–9.7) |
| Min–max | 0.2–15.5 | 0.9–18.8 | 0.2–18.8 |
| Sex (%) | |||
| Female | 14 (50) | 33 (52) | 47 (52) |
| Age at onset (mo) | |||
| Median (IQR) | 3 (2–4) | 12 (8–18) | 8 (4–12) |
| Min–max | 0.5–7 | 3–96 | 0.5–96 |
| SMN2 copy number (%) | |||
| n | 27 (96) | 63 (100) | 90 (99) |
| 2 | 20 (71) | 1 (2) | 21 (23) |
| 3 | 7 (25) | 53 (84) | 60 (66) |
| 4 | 0 (0) | 8 (13) | 8 (9) |
| 5 | 0 (0) | 1 (2) | 1 (1) |
| NIV at visit (%) | |||
| n | 27 (96) | — | — |
| Yes | 13 (46) | — | — |
| No | 14 (50) | — | — |
| Feeding support at visit (%) | |||
| n | 27 (96) | 53 (84) | 80 (88) |
| Yes | 17 (61) | 7 (11) | 24 (26) |
| No | 10 (36) | 46 (73) | 56 (62) |
Note: Number of patients (n) is stated if data were incomplete. Limited information (n = 1) about NIV in patients with Types 2/3 SMA was available and was therefore not presented in this table (dashes). Median (IQR) and min–max were reported, because variables were not normally distributed in at least one stratum.